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1

Oh, Youngbin, Hyeonjin Kim, and Sang-Gyu Kim. "Virus-induced plant genome editing." Current Opinion in Plant Biology 60 (April 2021): 101992. http://dx.doi.org/10.1016/j.pbi.2020.101992.

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Zhang, Chao, Shanhe Liu, Xuan Li, Ruixuan Zhang, and Jun Li. "Virus-Induced Gene Editing and Its Applications in Plants." International Journal of Molecular Sciences 23, no. 18 (2022): 10202. http://dx.doi.org/10.3390/ijms231810202.

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CRISPR/Cas-based genome editing technologies, which allow the precise manipulation of plant genomes, have revolutionized plant science and enabled the creation of germplasms with beneficial traits. In order to apply these technologies, CRISPR/Cas reagents must be delivered into plant cells; however, this is limited by tissue culture challenges. Recently, viral vectors have been used to deliver CRISPR/Cas reagents into plant cells. Virus-induced genome editing (VIGE) has emerged as a powerful method with several advantages, including high editing efficiency and a simplified process for generati
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3

Uranga, Mireia, Marta Vazquez-Vilar, Diego Orzáez, and José-Antonio Darós. "CRISPR-Cas12a Genome Editing at the Whole-Plant Level Using Two Compatible RNA Virus Vectors." CRISPR Journal 4, no. 5 (2021): 761–69. https://doi.org/10.1089/crispr.2021.0049.

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The use of viral vectors that can replicate and move systemically through the host plant to deliver bacterialCRISPR components enables genome editing at the whole-plant level and avoids the requirement for labor-intensive stable transformation. However, this approach usually relies on previously transformed plants thatstably express a CRISPR-Cas nuclease. Here, we describe successful DNA-free genome editing ofNicotiana ben-thamianausing two compatible RNA virus vectors derived from tobacco etch virus (TEV; genusPotyvirus) andpotato virus X (PVX; genusPotexvirus), which replicate in the same ce
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Uranga, Mireia, Verónica Aragonés, Sara Selma, Marta Vázquez-Vilar, Diego Orzáez, and José-Antonio Darós. "Efficient Cas9 multiplex editing using unspaced sgRNA arrays engineering in a Potato virus X vector." Plant Journal 106, no. 2 (2021): 555–65. https://doi.org/10.1111/tpj.15164.

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Systems based on the clustered, regularly interspaced, short palindromic repeat (CRISPR) and CRISPR-associated proteins (Cas) have revolutionized genome editing in many organisms, including plants. Most CRISPR-Cas strategies in plants rely on genetic transformation using&nbsp;<em>Agrobacterium tumefaciens</em>&nbsp;to supply the gene editing reagents, such as Cas nucleases or the synthetic guide RNA (sgRNA). While Cas nucleases are constant elements in editing approaches, sgRNAs are target-specific and a screening process is usually required to identify those most effective. Plant virus-derive
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5

Mikhaylova, Elena. "Virus-Induced Genome Editing (VIGE): One Step Away from an Agricultural Revolution." International Journal of Molecular Sciences 26, no. 10 (2025): 4599. https://doi.org/10.3390/ijms26104599.

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There is currently a worldwide trend towards deregulating the use of genome-edited plants. Virus-induced genome editing (VIGE) is a novel technique that utilizes viral vectors to transiently deliver clustered regularly interspaced short palindromic repeat (CRISPR) components into plant cells. It potentially allows us to obtain transgene-free events in any plant species in a single generation without in vitro tissue culture. This technology has great potential for agriculture and is already being applied to more than 14 plant species using more than 20 viruses. The main limitations of VIGE incl
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6

Ghoshal, Basudev, Brandon Vong, Colette L. Picard, Suhua Feng, Janet M. Tam, and Steven E. Jacobsen. "A viral guide RNA delivery system for CRISPR-based transcriptional activation and heritable targeted DNA demethylation in Arabidopsis thaliana." PLOS Genetics 16, no. 12 (2020): e1008983. http://dx.doi.org/10.1371/journal.pgen.1008983.

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Plant RNA viruses are used as delivery vectors for their high level of accumulation and efficient spread during virus multiplication and movement. Utilizing this concept, several viral-based guide RNA delivery platforms for CRISPR-Cas9 genome editing have been developed. The CRISPR-Cas9 system has also been adapted for epigenome editing. While systems have been developed for CRISPR-Cas9 based gene activation or site-specific DNA demethylation, viral delivery of guide RNAs remains to be developed for these purposes. To address this gap we have developed a tobacco rattle virus (TRV)-based single
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7

Fondong, Vincent N., Ugrappa Nagalakshmi, and Savithramma P. Dinesh-Kumar. "Novel Functional Genomics Approaches: A Promising Future in the Combat Against Plant Viruses." Phytopathology® 106, no. 10 (2016): 1231–39. http://dx.doi.org/10.1094/phyto-03-16-0145-fi.

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Advances in functional genomics and genome editing approaches have provided new opportunities and potential to accelerate plant virus control efforts through modification of host and viral genomes in a precise and predictable manner. Here, we discuss application of RNA-based technologies, including artificial micro RNA, transacting small interfering RNA, and Cas9 (clustered regularly interspaced short palindromic repeat–associated protein 9), which are currently being successfully deployed in generating virus-resistant plants. We further discuss the reverse genetics approach, targeting induced
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8

Surya Krishna, Sakthivel, S. R. Harish Chandar, Maruthachalam Ravi, et al. "Transgene-Free Genome Editing for Biotic and Abiotic Stress Resistance in Sugarcane: Prospects and Challenges." Agronomy 13, no. 4 (2023): 1000. http://dx.doi.org/10.3390/agronomy13041000.

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Sugarcane (Saccharum spp.) is one of the most valuable food and industrial crops. Its production is constrained due to major biotic (fungi, bacteria, viruses and insect pests) and abiotic (drought, salt, cold/heat, water logging and heavy metals) stresses. The ever-increasing demand for sugar and biofuel and the rise of new pest and disease variants call for the use of innovative technologies to speed up the sugarcane genetic improvement process. Developing new cultivars through conventional breeding techniques requires much time and resources. The advent of CRISPR/Cas genome editing technolog
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9

Akhmetollayeva, A. S., and Sh A. Manabayeva. "CREATION OF AN EXPRESSION VECTOR FOR MULTIPLEX EDITING OF THE POTATO VACUOLAR INVERTASE GENE USING THE CRISPR/CAS9 SYSTEM." Eurasian Journal of Applied Biotechnology, no. 3 (October 3, 2024): 43–55. http://dx.doi.org/10.11134/btp.3.2024.5.

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Creating genetic engineering constructs for plant genome editing requires considerable time, material resources, and specialized equipment. A thorough understanding of the functions and efficacy of each construct element is critical to the design of specialized vectors for specific tasks. This article provides a detailed overview of the process of creating genetic engineering constructs for editing the invertase gene responsible for cold-induced sweetening (CIS) in potato tubers, starting with the design of the variable part of the guide RNA and ending with the assembly of the final expression
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10

Matoušková, Magda, Jiří Plachý, Dana Kučerová, et al. "Rapid adaptive evolution of avian leukosis virus subgroup J in response to biotechnologically induced host resistance." PLOS Pathogens 20, no. 8 (2024): e1012468. http://dx.doi.org/10.1371/journal.ppat.1012468.

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Genetic editing of the germline using CRISPR/Cas9 technology has made it possible to alter livestock traits, including the creation of resistance to viral diseases. However, virus adaptability could present a major obstacle in this effort. Recently, chickens resistant to avian leukosis virus subgroup J (ALV-J) were developed by deleting a single amino acid, W38, within the ALV-J receptor NHE1 using CRISPR/Cas9 genome editing. This resistance was confirmed both in vitro and in vivo. In vitro resistance of W38-/- chicken embryonic fibroblasts to all tested ALV-J strains was shown. To investigate
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11

Jo, Areum, Sangwoo Ham, Gum Hwa Lee, et al. "Efficient Mitochondrial Genome Editing by CRISPR/Cas9." BioMed Research International 2015 (2015): 1–10. http://dx.doi.org/10.1155/2015/305716.

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The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 system has been widely used for nuclear DNA editing to generate mutations or correct specific disease alleles. Despite its flexible application, it has not been determined if CRISPR/Cas9, originally identified as a bacterial defense system against virus, can be targeted to mitochondria for mtDNA editing. Here, we show that regular FLAG-Cas9 can localize to mitochondria to edit mitochondrial DNA with sgRNAs targeting specific loci of the mitochondrial genome. Expression of FLAG-Cas9 together with gRNA targeting Cox1 and
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12

Chupradit, Koollawat, Nontaphat Thongsin, Chatchai Tayapiwatana, and Methichit Wattanapanitch. "A precise gene delivery approach for human induced pluripotent stem cells using Cas9 RNP complex and recombinant AAV6 donor vectors." PLOS ONE 17, no. 7 (2022): e0270963. http://dx.doi.org/10.1371/journal.pone.0270963.

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Genome editing in human induced pluripotent stem cells (hiPSCs) offers a potential tool for studying gene functions in disease models and correcting genetic mutations for cell-based therapy. Precise transgene insertion in hiPSCs represents a significant challenge. In the past decade, viral transduction has been widely used due to its high transduction efficiency; however, it can result in random transgene integration and variable transgene copy numbers. Non-viral-based strategies are generally safer but limited by their low transfection efficiency in hiPSCs. Recently, genome engineering using
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13

Zamai, Loris. "Unveiling Human Non-Random Genome Editing Mechanisms Activated in Response to Chronic Environmental Changes: I. Where Might These Mechanisms Come from and What Might They Have Led To?" Cells 9, no. 11 (2020): 2362. http://dx.doi.org/10.3390/cells9112362.

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This article challenges the notion of the randomness of mutations in eukaryotic cells by unveiling stress-induced human non-random genome editing mechanisms. To account for the existence of such mechanisms, I have developed molecular concepts of the cell environment and cell environmental stressors and, making use of a large quantity of published data, hypothesised the origin of some crucial biological leaps along the evolutionary path of life on Earth under the pressure of natural selection, in particular, (1) virus–cell mating as a primordial form of sexual recombination and symbiosis; (2) L
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14

Chemello, F., A. C. Chai, H. Li, et al. "Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing." Science Advances 7, no. 18 (2021): eabg4910. http://dx.doi.org/10.1126/sciadv.abg4910.

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Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of dystrophin, which maintains muscle membrane integrity. We used an adenine base editor (ABE) to modify splice donor sites of the dystrophin gene, causing skipping of a common DMD deletion mutation of exon 51 (∆Ex51) in cardiomyocytes derived from human induced pluripotent stem cells, restoring dystrophin expression. Prime editing was also capable of reframing the dystrophin open reading frame in these cardiomyocytes. Intramuscular injection of ∆Ex51 mice with adeno-associated virus serotype-9 encoding ABE componen
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15

Dev, Kapil, Jubeda Begum, Nasir Akbar Mir, and Rajiv Kant. "Advancements of CRISPR/Cas9 technology and its value in antiviral therapeutics." Letters In Animal Biology 1, no. 1 (2021): 46–57. http://dx.doi.org/10.62310/liab.v1i1.60.

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CISPR/Cas9 system is a natural immune mechanism adopted by bacteria and archaea on exposure to invading phages and plasmids. The field of genome editing has been revolutionized with the advent of CRISPR/Cas9 technology. The CRISPR/Cas9 based gene editing has offered a promising therapeutic platform for many animal and human diseases, particularly viral diseases because viruses evolve constantly and hence escape vaccine-induced immunity. The targeted genome editing by RNA-guided nucleases is rapid, easy, economical, and efficient compared to previous editing technologies. It not only helps in t
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16

Koslová, Anna, Dana Kučerová, Markéta Reinišová, Josef Geryk, Pavel Trefil, and Jiří Hejnar. "Genetic Resistance to Avian Leukosis Viruses Induced by CRISPR/Cas9 Editing of Specific Receptor Genes in Chicken Cells." Viruses 10, no. 11 (2018): 605. http://dx.doi.org/10.3390/v10110605.

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Avian leukosis viruses (ALVs), which are pathogens of concern in domestic poultry, utilize specific receptor proteins for cell entry that are both necessary and sufficient for host susceptibility to a given ALV subgroup. This unequivocal relationship offers receptors as suitable targets of selection and biotechnological manipulation with the aim of obtaining virus-resistant poultry. This approach is further supported by the existence of natural knock-outs of receptor genes that segregate in inbred lines of chickens. We used CRISPR/Cas9 genome editing tools to introduce frame-shifting indel mut
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17

Phuphuakrat, Angsana, Romchat Kraiwong, Chompunuch Boonarkart, Darat Lauhakirti, Tun-Hou Lee, and Prasert Auewarakul. "Double-Stranded RNA Adenosine Deaminases Enhance Expression of Human Immunodeficiency Virus Type 1 Proteins." Journal of Virology 82, no. 21 (2008): 10864–72. http://dx.doi.org/10.1128/jvi.00238-08.

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ABSTRACT ADARs (adenosine deaminases that act on double-stranded RNA) are RNA editing enzymes that catalyze a change from adenosine to inosine, which is then recognized as guanosine by translational machinery. We demonstrate here that overexpression of ADARs but not of an ADAR mutant lacking editing activity could upregulate human immunodeficiency virus type 1 (HIV-1) structural protein expression and viral production. Knockdown of ADAR1 by RNA silencing inhibited HIV-1 production. Viral RNA harvested from transfected ADAR1-knocked-down cells showed a decrease in the level of unspliced RNA tra
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18

Zhang, Rui-Xiang, Yun-Fei Zhang, Hao Yang, et al. "An Optimized Editing Approach for Wheat Genes by Improving sgRNA Design and Transformation Strategies." International Journal of Molecular Sciences 26, no. 8 (2025): 3796. https://doi.org/10.3390/ijms26083796.

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Hexaploid wheat has a large genome, making it difficult for transgenes to produce phenotypes due to gene redundancy and tight linkage among genes. Multiple gene copies typically necessitate multiple targeting events during gene editing, followed by several generations of self-crossing to achieve homozygous genotypes. The high cost of transgenesis in wheat is another issue, which hinders the easy availability of gene-edited materials in wheat. In this study, we developed a comprehensive approach to improve wheat gene editing efficiency. First, we established a protoplast-based system to evaluat
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19

Oh, Youngbin, and Sang-Gyu Kim. "RPS5A Promoter-Driven Cas9 Produces Heritable Virus-Induced Genome Editing in Nicotiana attenuata." Molecules and Cells 44, no. 12 (2021): 911–19. http://dx.doi.org/10.14348/molcells.2021.0237.

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20

Shin, Hye Jin, Keun Bon Ku, Soojin Kim, et al. "A Crucial Role of ACBD3 Required for Coxsackievirus Infection in Animal Model Developed by AAV-Mediated CRISPR Genome Editing Technique." Viruses 13, no. 2 (2021): 237. http://dx.doi.org/10.3390/v13020237.

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Genetic screens using CRISPR/Cas9 have been exploited to discover host–virus interactions. These screens have identified viral dependencies on host proteins during their life cycle and potential antiviral strategies. The acyl-CoA binding domain containing 3 (ACBD3) was identified as an essential host factor for the Coxsackievirus B3 (CVB3) infection. Other groups have also investigated the role of ACBD3 as a host factor for diverse enteroviruses in cultured cells. However, it has not been tested if ACBD3 is required in the animal model of CVB3 infection. Owing to embryonic lethality, conventio
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21

Kim, Jihoon, Bon-Kyoung Koo, and Ki-Jun Yoon. "Modeling Host-Virus Interactions in Viral Infectious Diseases Using Stem-Cell-Derived Systems and CRISPR/Cas9 Technology." Viruses 11, no. 2 (2019): 124. http://dx.doi.org/10.3390/v11020124.

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Pathologies induced by viral infections have undergone extensive study, with traditional model systems such as two-dimensional (2D) cell cultures and in vivo mouse models contributing greatly to our understanding of host-virus interactions. However, the technical limitations inherent in these systems have constrained efforts to more fully understand such interactions, leading to a search for alternative in vitro systems that accurately recreate in vivo physiology in order to advance the study of viral pathogenesis. Over the last decade, there have been significant technological advances that h
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22

Langevin, Christelle, Pierre Boudinot, and Bertrand Collet. "IFN Signaling in Inflammation and Viral Infections: New Insights from Fish Models." Viruses 11, no. 3 (2019): 302. http://dx.doi.org/10.3390/v11030302.

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The overarching structure of the type I interferon (IFN) system is conserved across vertebrates. However, the variable numbers of whole genome duplication events during fish evolution offer opportunities for the expansion, diversification, and new functionalization of the genes that are involved in antiviral immunity. In this review, we examine how fish models provide new insights about the implication of virus-driven inflammation in immunity and hematopoiesis. Mechanisms that have been discovered in fish, such as the strong adjuvant effect of type I IFN that is used with DNA vaccination, cons
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23

Brauer, Elizabeth K., Margaret Balcerzak, Hélène Rocheleau, et al. "Genome Editing of a Deoxynivalenol-Induced Transcription Factor Confers Resistance to Fusarium graminearum in Wheat." Molecular Plant-Microbe Interactions® 33, no. 3 (2020): 553–60. http://dx.doi.org/10.1094/mpmi-11-19-0332-r.

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Deoxynivalenol (DON) is a mycotoxin virulence factor that promotes growth of the Fusarium graminearum fungus in wheat floral tissues. To further our understanding of the effects of DON exposure on plant cell function, we characterized DON-induced transcriptional changes in wheat spikelets. Four hundred wheat genes were differentially expressed during infection with wild-type F. graminearum as compared with a Δtri5 mutant strain that is unable to produce DON. Most of these genes were more induced by the DON-producing strain and included genes involved in secondary metabolism, signaling, transpo
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24

Hahn, Florian, Laura Sanjurjo Loures, Caroline A. Sparks, Kostya Kanyuka, and Vladimir Nekrasov. "Efficient CRISPR/Cas-Mediated Targeted Mutagenesis in Spring and Winter Wheat Varieties." Plants 10, no. 7 (2021): 1481. http://dx.doi.org/10.3390/plants10071481.

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CRISPR/Cas technology has recently become the molecular tool of choice for gene function studies in plants as well as crop improvement. Wheat is a globally important staple crop with a well annotated genome and there is plenty of scope for improving its agriculturally important traits using genome editing technologies, such as CRISPR/Cas. As part of this study we targeted three different genes in hexaploid wheat Triticum aestivum: TaBAK1-2 in the spring cultivar Cadenza as well as Ta-eIF4E and Ta-eIF(iso)4E in winter cultivars Cezanne, Goncourt and Prevert. Primary transgenic lines carrying CR
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25

Gandy, Sharon Z., Sarah D. Linnstaedt, Sumitra Muralidhar, Kathleen A. Cashman, Leonard J. Rosenthal, and John L. Casey. "RNA Editing of the Human Herpesvirus 8 Kaposin Transcript Eliminates Its Transforming Activity and Is Induced during Lytic Replication." Journal of Virology 81, no. 24 (2007): 13544–51. http://dx.doi.org/10.1128/jvi.01521-07.

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ABSTRACT Human herpesvirus 8 is the etiologic agent associated with Kaposi's sarcoma and primary effusion lymphoma (PEL). The K12 RNA, which produces as many as three variants of the kaposin protein, as well as a microRNA, is the most abundant transcript expressed in latent Kaposi's sarcoma-associated herpesvirus infection, and yet it is also induced during lytic replication. The portion of the transcript that includes the microRNA and the kaposin A sequence has been shown to have tumorigenic potential. Genome coordinate 117990, which is within this transcript, has been found to be heterogeneo
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26

Zahn, Roland C., Ina Schelp, Olaf Utermöhlen, and Dorothee von Laer. "A-to-G Hypermutation in the Genome of Lymphocytic Choriomeningitis Virus." Journal of Virology 81, no. 2 (2006): 457–64. http://dx.doi.org/10.1128/jvi.00067-06.

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ABSTRACT The interferon-inducible adenosine deaminase that acts on double-stranded RNA (ADAR1-L) has been proposed to be one of the antiviral effector proteins within the complex innate immune response. Here, the potential role of ADAR1-L in the innate immune response to lymphocytic choriomeningitis virus (LCMV), a widely used virus model, was studied. Infection with LCMV clearly upregulated ADAR1-L expression and activity. The editing activity of ADAR1-L on an RNA substrate was not inhibited by LCMV replication. Accordingly, an adenosine-to-guanosine (A-to-G) and uracil-to-cytidine (U-to-C) h
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27

Faden, Daniel L., Krystle A. Lang Kuhs, Maoxuan Lin, et al. "APOBEC Mutagenesis Is Concordant between Tumor and Viral Genomes in HPV-Positive Head and Neck Squamous Cell Carcinoma." Viruses 13, no. 8 (2021): 1666. http://dx.doi.org/10.3390/v13081666.

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APOBEC is a mutagenic source in human papillomavirus (HPV)-mediated malignancies, including HPV+ oropharyngeal squamous cell carcinoma (HPV + OPSCC), and in HPV genomes. It is unknown why APOBEC mutations predominate in HPV + OPSCC, or if the APOBEC-induced mutations observed in both human cancers and HPV genomes are directly linked. We performed sequencing of host somatic exomes, transcriptomes, and HPV16 genomes from 79 HPV + OPSCC samples, quantifying APOBEC mutational burden and activity in both host and virus. APOBEC was the dominant mutational signature in somatic exomes. In viral genome
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28

Jiang, David J., Christine L. Xu, and Stephen H. Tsang. "Revolution in Gene Medicine Therapy and Genome Surgery." Genes 9, no. 12 (2018): 575. http://dx.doi.org/10.3390/genes9120575.

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Recently, there have been revolutions in the development of both gene medicine therapy and genome surgical treatments for inherited disorders. Much of this progress has been centered on hereditary retinal dystrophies, because the eye is an immune-privileged and anatomically ideal target. Gene therapy treatments, already demonstrated to be safe and efficacious in numerous clinical trials, are benefitting from the development of new viral vectors, such as dual and triple adeno-associated virus (AAV) vectors. CRISPR/Cas9, which revolutionized the field of gene editing, is being adapted into more
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Hammad, Ruba, Jamal Alzubi, Manuel Rhiel, et al. "CRISPR-Cas12a for Highly Efficient and Marker-Free Targeted Integration in Human Pluripotent Stem Cells." International Journal of Molecular Sciences 25, no. 2 (2024): 985. http://dx.doi.org/10.3390/ijms25020985.

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The CRISPR-Cas12a platform has attracted interest in the genome editing community because the prototypical Acidaminococcus Cas12a generates a staggered DNA double-strand break upon binding to an AT-rich protospacer-adjacent motif (PAM, 5′-TTTV). The broad application of the platform in primary human cells was enabled by the development of an engineered version of the natural Cas12a protein, called Cas12a Ultra. In this study, we confirmed that CRISPR-Cas12a Ultra ribonucleoprotein complexes enabled allelic gene disruption frequencies of over 90% at multiple target sites in human T cells, hemat
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30

Kruglova, N. A., D. S. Komkov, D. V. Mazurov, and M. V. Shepelev. "The RRE-REV module has no effect on the packaging efficiency of cas9 and Gag proteins into nanomedic virus-like particles." Доклады Российской академии наук. Науки о жизни 515, no. 2 (2024): 64–70. http://dx.doi.org/10.31857/s2686738924020121.

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Delivery of ribonucleoprotein complexes of Cas9 nuclease and guide RNA into target cells with virus-like particles (VLP) is one of the novel methods of genome editing, suitable for gene therapy of human diseases in the future. Efficiency of genome editing with VLPs depends on the packaging of Cas9 into VLPs, that is mediated by viral Gag protein. To increase the packaging of Cas9 into NanoMEDIC system VLPs plasmid constructs for expression of Cas9 and Gag were modified by the addition of HIV RRE (Rev response element), that is expected to increase the nuclear export of RRE-containing transcrip
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31

Venkatesh, Jelli, Seo-Young Lee, Hwa-Jeong Kang, Seyoung Lee, Joung-Ho Lee, and Byoung-Cheorl Kang. "Heat Stress Induced Potato virus X-mediated CRISPR/Cas9 Genome Editing in Nicotiana benthamiana." Plant Breeding and Biotechnology 10, no. 3 (2022): 186–96. http://dx.doi.org/10.9787/pbb.2022.10.3.186.

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32

Temblador, Arturo, Dimitrios Topalis, Graciela Andrei, and Robert Snoeck. "CRISPR/Cas9 Editing of the Polyomavirus Tumor Antigens Inhibits Merkel Cell Carcinoma Growth In Vitro." Cancers 11, no. 9 (2019): 1260. http://dx.doi.org/10.3390/cancers11091260.

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Merkel cell carcinoma (MCC) is an aggressive type of skin cancer whose main causative agent is Merkel cell polyomavirus (MCPyV). MCPyV is integrated into the genome of the tumor cells in most MCCs. Virus-positive tumor cells constitutively express two viral oncoproteins that promote cell growth: the small (sT) and the large (LT) tumor antigens (TAs). Despite the success of immunotherapies in patients with MCC, not all individuals respond to these treatments. Therefore, new therapeutic options continue to be investigated. Herein, we used CRISPR/Cas9 to target the viral oncogenes in two virus-po
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Caron, Jérôme, Véronique Pène, Laia Tolosa, et al. "Low-density lipoprotein receptor-deficient hepatocytes differentiated from induced pluripotent stem cells allow familial hypercholesterolemia modeling, CRISPR/Cas-mediated genetic correction, and productive hepatitis C virus infection." Stem Cell Research & Therapy 10, no. 1 (2019): 221. https://doi.org/10.1186/s13287-019-1342-6.

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<strong>Background: </strong>Familial hypercholesterolemia type IIA (FH) is due to mutations in the low-density lipoprotein receptor (LDLR) resulting in elevated levels of low-density lipoprotein cholesterol (LDL-c) in plasma and in premature cardiovascular diseases. As hepatocytes are the only cells capable of metabolizing cholesterol, they are therefore the target cells for cell/gene therapy approaches in the treatment of lipid metabolism disorders. Furthermore, the LDLR has been reported to be involved in hepatitis C virus (HCV) entry into hepatocytes; however, its role in the virus infecti
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34

Acha, Giovana, Ricardo Vergara, Marisol Muñoz, et al. "A Traceable DNA-Replicon Derived Vector to Speed Up Gene Editing in Potato: Interrupting Genes Related to Undesirable Postharvest Tuber Traits as an Example." Plants 10, no. 9 (2021): 1882. http://dx.doi.org/10.3390/plants10091882.

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In potato (Solanum tuberosum L.), protoplast techniques are limited to a few genotypes; thus, the use of regular regeneration procedures of multicellular explants causes us to face complexities associated to CRISPR/Cas9 gene editing efficiency and final identification of individuals. Geminivirus-based replicons contained in T-DNAs could provide an improvement to these procedures considering their cargo capability. We built a Bean yellow dwarf virus-derived replicon vector, pGEF-U, that expresses all the editing reagents under a multi-guide RNA condition, and the Green Fluorescent Protein (GFP)
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35

Chakraborty, Sandeep. "Sequencing data from Massachusetts General Hospital shows Cas9 integration into the genome, highlighting a serious hazard in gene-editing therapeutics." F1000Research 8 (November 4, 2019): 1846. http://dx.doi.org/10.12688/f1000research.20744.1.

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The ability to edit a specific gene within our genomes using guided-nucleases (Cas9/ZFN/TALEN - CaZiTa) presents huge opportunities for curing many genetic disorders. Delivery of this ‘drug’ within cells is a critical step for such therapies. The ability of recombinant adeno-associated virus (rAAV) to enter cells makes it a perfect choice as a vector for gene therapy. A plasmid comprising the rAAV, the CaZiTa, guide RNAs (for CRISPR) is expected to enter the cell, edit the target gene(s), remain episomal, and thus fade away with time. However, the rather obvious danger of integration of the pl
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Wu, Guize, Yunhua Ding, Ning Li, Hongji Zhang, and Ning Liu. "Genome-Wide Identification of the Sulfate Transporter Gene Family Reveals That BolSULTR2;1 Regulates Plant Resistance to Alternaria brassicicola Through the Modulation of Glutathione Biosynthesis in Broccoli." Antioxidants 14, no. 4 (2025): 496. https://doi.org/10.3390/antiox14040496.

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Sulfate transporters (SULTRs) are key players that regulate sulfur acquisition and distribution within plants, thereby influencing cellular redox hemostasis under pathogen attacks, such as Alternaria brassicicola (Ab). In this study, a total of 23 BolSULTR (Brassica oleracea SULTR) genes were identified from the Brassica genome. These BolSULTRs are distributed across nine chromosomes, with all collinear BolSULTR gene pairs undergoing purifying selections. Phylogenetic analysis reveals that the SULTR family is evolutionarily conserved among plant kingdoms. qRT-PCR analysis demonstrated that the
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Iwasawa, Chizuru, Ryota Tamura, Yuki Sugiura, et al. "Increased Cytotoxicity of Herpes Simplex Virus Thymidine Kinase Expression in Human Induced Pluripotent Stem Cells." International Journal of Molecular Sciences 20, no. 4 (2019): 810. http://dx.doi.org/10.3390/ijms20040810.

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Human induced pluripotent stem cells (iPSCs) hold enormous promise for regenerative medicine. The major safety concern is the tumorigenicity of transplanted cells derived from iPSCs. A potential solution would be to introduce a suicide gene into iPSCs as a safety switch. The herpes simplex virus type 1 thymidine kinase (HSV-TK) gene, in combination with ganciclovir, is the most widely used enzyme/prodrug suicide system from basic research to clinical applications. In the present study, we attempted to establish human iPSCs that stably expressed HSV-TK with either lentiviral vectors or CRISPR/C
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Wichmann, Max, Cecile L. Maire, Niklas Nuppenau, et al. "Deep Characterization and Comparison of Different Retrovirus-like Particles Preloaded with CRISPR/Cas9 RNPs." International Journal of Molecular Sciences 24, no. 14 (2023): 11399. http://dx.doi.org/10.3390/ijms241411399.

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The CRISPR/Cas system has a broad range of possible medical applications, but its clinical translation has been hampered, particularly by the lack of safe and efficient vector systems mediating the short-term expression of its components. Recently, different virus-like particles (VLPs) have been introduced as promising vectors for the delivery of CRISPR/Cas genome editing components. Here, we characterized and directly compared three different types of retrovirus-based (R) VLPs, two derived from the γ-retrovirus murine leukemia virus (gRVLPs and “enhanced” egRVLPs) and one from the lentivirus
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39

Badia, Roger, Eduardo Pauls, Eva Riveira-Munoz, Bonaventura Clotet, José A. Esté, and Ester Ballana. "Zinc Finger Endonuclease TargetingPSIP1Inhibits HIV-1 Integration." Antimicrobial Agents and Chemotherapy 58, no. 8 (2014): 4318–27. http://dx.doi.org/10.1128/aac.02690-14.

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ABSTRACTGenome editing using zinc finger nucleases (ZFNs) has been successfully applied to disrupt CCR5 or CXCR4 host factors and inhibit viral entry and infection. Gene therapy using ZFNs to modify thePSIP1gene, which encodes the lens epithelium-derived growth factor (LEDGF) protein, might restrain an early step of the viral replication cycle at the integration level. ZFNs targeting thePSIP1gene (ZFNLEDGF) were designed to specifically recognize the sequence after the integrase binding domain (IBD) of the LEDGF/p75 protein. ZFNLEDGFsuccessfully recognized the target region of thePSIP1gene in
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Hamada, Taiji, Seiya Yokoyama, Toshiaki Akahane, et al. "Electroporation Induces Unexpected Alterations in Gene Expression: A Tip for Selection of Optimal Transfection Method." Current Issues in Molecular Biology 47, no. 2 (2025): 91. https://doi.org/10.3390/cimb47020091.

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Electroporation is an efficient method for nucleotide and protein transfer, and is used for clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein 9 (Cas9)-mediated genome editing. In this study, we investigated the effects of electroporation on platelet-derived growth factor receptor alpha (PDGFRA) and receptor tyrosine kinase (RTK) expression in U-251 and U-87 MG cells. PDGFRA mRNA and protein expression decreased 2 days after electroporation in both cell lines, with recovery observed after 13 days in U-87 MG cells. However, in U-251 MG cells, PDGFRα expression
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moghadam, Mohadeseh Soleimanpour, Azad Khosh, and Diako Ebrahimi. "Abstract 3401: Uncovering virus induced dysregulated processes in cancer and their impact on outcomes." Cancer Research 84, no. 6_Supplement (2024): 3401. http://dx.doi.org/10.1158/1538-7445.am2024-3401.

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Abstract The National Cancer Institute has designated several viruses such as EBV, HBV, HCV, HIV, HPV, and HTLV as agents that either cause cancer or increase the risk of developing cancer. However, extensive analyses of tumor genome and transcriptome datasets have identified numerous additional virus types, the functional implications of which remain largely unknown. Viral infection often dysregulates unique molecular processes within tumor cells and alters the composition of immune cells in the tumor microenvironment. For instance, HPV-positive tumors have been shown to exhibit a significant
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Malur, Achut G., Santanu Chattopadhyay, Ratan K. Maitra, and Amiya K. Banerjee. "Inhibition of STAT 1 Phosphorylation by Human Parainfluenza Virus Type 3 C Protein." Journal of Virology 79, no. 12 (2005): 7877–82. http://dx.doi.org/10.1128/jvi.79.12.7877-7882.2005.

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ABSTRACT The P mRNA of the viruses belonging to the subfamily Paramyxovirinae possesses a unique property of giving rise to several accessory proteins by a process that involves the utilization of overlapping open reading frames (the C proteins) and by an “RNA-editing” mechanism (the V proteins). Although these proteins are considered accessory, numerous studies have highlighted the importance of these proteins in virus transcription and interferon signaling, including our previous observation on the role of human parainfluenza virus type 3 (HPIV 3) C protein in the transcription of viral geno
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Wang, Yaxin, Naeem Zafar, Qurban Ali, et al. "CRISPR/Cas Genome Editing Technologies for Plant Improvement against Biotic and Abiotic Stresses: Advances, Limitations, and Future Perspectives." Cells 11, no. 23 (2022): 3928. http://dx.doi.org/10.3390/cells11233928.

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Crossbreeding, mutation breeding, and traditional transgenic breeding take much time to improve desirable characters/traits. CRISPR/Cas-mediated genome editing (GE) is a game-changing tool that can create variation in desired traits, such as biotic and abiotic resistance, increase quality and yield in less time with easy applications, high efficiency, and low cost in producing the targeted edits for rapid improvement of crop plants. Plant pathogens and the severe environment cause considerable crop losses worldwide. GE approaches have emerged and opened new doors for breeding multiple-resistan
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Sun, Haotian. "The Challenge Facing CRISPR/Cas9 System: Off-Target Effects and Their Optimization." Highlights in Science, Engineering and Technology 74 (December 29, 2023): 782–87. http://dx.doi.org/10.54097/psd28z73.

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The field of genome editing has undergone a profound revolution with the emergence of CRISPR-Cas9 technology, which enables precise modifications to the genetic code. However, a critical concern is the possibility for the appearance of off-target, where the modifications induced by Cas9 nuclease are at non-intended targets. The mismatches of the seed sequence with the single guide RNA (sgRNA) and the inappropriate length of it could induce off-target effects. Moreover, the inflammatory response triggered by virus-mediated delivery methods may also be responsible for off-target. For the expecte
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Mehta, Anuradha, Yoshinori Shirai, Emi Kouyama-Suzuki, et al. "IQSEC2 Deficiency Results in Abnormal Social Behaviors Relevant to Autism by Affecting Functions of Neural Circuits in the Medial Prefrontal Cortex." Cells 10, no. 10 (2021): 2724. http://dx.doi.org/10.3390/cells10102724.

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IQSEC2 is a guanine nucleotide exchange factor (GEF) for ADP-ribosylation factor 6 (Arf6), of which protein is exclusively localized to the postsynaptic density of the excitatory synapse. Human genome studies have revealed that the IQSEC2 gene is associated with X-linked neurodevelopmental disorders, such as intellectual disability (ID), epilepsy, and autism. In this study, we examined the behavior and synapse function in IQSEC2 knockout (KO) mice that we generated using CRIPSR/Cas9-mediated genome editing to solve the relevance between IQSEC2 deficiency and the pathophysiology of neurodevelop
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Kumar, Prafulla, Sushmita, Ankit Agrawal, Abhay Kumar, and Ravindra Kumar. "Unveiling the Molecular Arsenal: NIK1-Mediated Translation Suppression as a Key Player in Plant Antiviral Immunity." Journal of Scientific Research and Reports 31, no. 4 (2025): 30–43. https://doi.org/10.9734/jsrr/2025/v31i42925.

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Plant cells are continually exposed to a variety of microbes, with viral infections standing out as a major agricultural challenge. Viruses often undermine plant defenses, posing a significant threat to the productivity of crucial crops and global food security. Plant viruses, due to their limited coding capacity, heavily rely on the host cell machinery during infection, interacting with numerous host proteins. Given the absence of translation-required components in viral genomes, plant viruses have evolved strategies to manipulate the host protein synthesis machinery for viral protein product
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Apinda, Nisachon, Anucha Muenthaisong, Paweena Chomjit, et al. "Simultaneous Protective Immune Responses of Ducks against Duck Plague and Fowl Cholera by Recombinant Duck Enteritis Virus Vector Expressing Pasteurella multocida OmpH Gene." Vaccines 10, no. 8 (2022): 1358. http://dx.doi.org/10.3390/vaccines10081358.

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Duck enteritis virus and Pasteurella multocida are major duck pathogens that induce duck plague and fowl cholera, respectively, in ducks and other waterfowl populations, leading to high levels of morbidity and mortality. Immunization with live attenuated DEV vaccine containing P. multocida outer membrane protein H (OmpH) can provide the most effective protection against these two infectious diseases in ducks. We have recently reported the construction of recombinant DEV expressing P. multocida ompH gene using the CRISPR/Cas9 gene editing strategy with the goal of using it as a bivalent vaccine
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48

Tolosa and Zhang. "The Role of Major Transcription Factors in Solanaceous Food Crops under Different Stress Conditions: Current and Future Perspectives." Plants 9, no. 1 (2020): 56. http://dx.doi.org/10.3390/plants9010056.

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Plant growth, development, and productivity are adversely affected by environmental stresses such as drought (osmotic stress), soil salinity, cold, oxidative stress, irradiation, and diverse diseases. These impacts are of increasing concern in light of climate change. Noticeably, plants have developed their adaptive mechanism to respond to environmental stresses by transcriptional activation of stress-responsive genes. Among the known transcription factors, DoF, WRKY, MYB, NAC, bZIP, ERF, ARF and HSF are those widely associated with abiotic and biotic stress response in plants. Genome-wide ide
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Moskalev, Alexander V., Boris Yu Gumilevsky, Vasiliy Ya Apchel, and Vasiliy N. Tsygan. "Features of the immunopathogenesis of infections caused by viroids and satellite viruses." Bulletin of the Russian Military Medical Academy 26, no. 2 (2024): 301–11. http://dx.doi.org/10.17816/brmma625370.

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One of the main molecular mechanisms of viroids that cause disease in plants is the blocking of ribonucleic acid functions of host cells by viroid ribonucleic acid. The variety of splicing options is due to viroid replication, which requires ribonucleic acid polymerase, ribonuclease, ligase, and deoxyribonucleic acid working on ribonucleic acid matrices. Viroids with faster replication are preferred. Identifying host proteins that interact with viroid ribonucleic acid is critical in the pathogenesis of viroid infections, which leads to gene expression changes of host plants. The study of the p
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Chang, Chia-Yu, Hsiao-Chien Ting, Ching-Ann Liu, et al. "Induced Pluripotent Stem Cell (iPSC)-Based Neurodegenerative Disease Models for Phenotype Recapitulation and Drug Screening." Molecules 25, no. 8 (2020): 2000. http://dx.doi.org/10.3390/molecules25082000.

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Neurodegenerative diseases represent a significant unmet medical need in our aging society. There are no effective treatments for most of these diseases, and we know comparatively little regarding pathogenic mechanisms. Among the challenges faced by those involved in developing therapeutic drugs for neurodegenerative diseases, the syndromes are often complex, and small animal models do not fully recapitulate the unique features of the human nervous system. Human induced pluripotent stem cells (iPSCs) are a novel technology that ideally would permit us to generate neuronal cells from individual
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