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Journal articles on the topic 'CRISPR, Gene editing, Parkinson, Gene therapy'

Author: Grafiati

Published: 14 March 2023

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1

Chung, Sun-Ku, and Seo-Young Lee. "Advances in Gene Therapy Techniques to Treat LRRK2 Gene Mutation." Biomolecules 12, no. 12 (2022): 1814. http://dx.doi.org/10.3390/biom12121814.

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Leucine-rich repeat kinase 2 (LRRK2) gene mutation is an autosomal dominant mutation associated with Parkinson’s disease (PD). Among LRRK2 gene mutations, the LRRK2 G2019S mutation is frequently involved in PD onset. Currently, diverse gene correction tools such as zinc finger nucleases (ZFNs), helper-dependent adenoviral vector (HDAdV), the bacterial artificial chromosome-based homologous recombination (BAC-based HR) system, and CRISPR/Cas9-homology-directed repair (HDR) or adenine base editor (ABE) are used in genome editing. Gene correction of the LRRK2 G2019S mutation has been applied when

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Rahman, Mujeeb ur, Muhammad Bilal, Junaid Ali Shah, Ajeet Kaushik, Pierre-Louis Teissedre, and Małgorzata Kujawska. "CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease." Pharmaceutics 14, no. 6 (2022): 1252. http://dx.doi.org/10.3390/pharmaceutics14061252.

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Parkinson’s disease (PD) and other chronic and debilitating neurodegenerative diseases (NDs) impose a substantial medical, emotional, and financial burden on individuals and society. The origin of PD is unknown due to a complex combination of hereditary and environmental risk factors. However, over the last several decades, a significant amount of available data from clinical and experimental studies has implicated neuroinflammation, oxidative stress, dysregulated protein degradation, and mitochondrial dysfunction as the primary causes of PD neurodegeneration. The new gene-editing techniques h

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De Plano, Laura M., Giovanna Calabrese, Sabrina Conoci, Salvatore P. P. Guglielmino, Salvatore Oddo, and Antonella Caccamo. "Applications of CRISPR-Cas9 in Alzheimer’s Disease and Related Disorders." International Journal of Molecular Sciences 23, no. 15 (2022): 8714. http://dx.doi.org/10.3390/ijms23158714.

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Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis, and Huntington’s disease represent some of the most prevalent neurodegenerative disorders afflicting millions of people worldwide. Unfortunately, there is a lack of efficacious treatments to cure or stop the progression of these disorders. While the causes of such a lack of therapies can be attributed to various reasons, the disappointing results of recent clinical trials suggest the need for novel and innovative approaches. Since its discovery, there has been a growing excitement around the potential for CRISPR-Cas9 medi

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Katzmann, Julius L., Arjen J. Cupido, and Ulrich Laufs. "Gene Therapy Targeting PCSK9." Metabolites 12, no. 1 (2022): 70. http://dx.doi.org/10.3390/metabo12010070.

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The last decades of research in cardiovascular prevention have been characterized by successful bench-to-bedside developments for the treatment of low-density lipoprotein (LDL) hypercholesterolemia. Recent examples include the inhibition of proprotein convertase subtilisin/kexin type 9 (PCSK9) with monoclonal antibodies, small interfering RNA and antisense RNA drugs. The cumulative effects of LDL cholesterol on atherosclerosis make early, potent, and long-term reductions in LDL cholesterol desirable—ideally without the need of regular intake or application of medication and importantly, withou

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Tang, Xuanting. "CRISPR/Cas9-based genome engineering in HIV gene therapy." E3S Web of Conferences 233 (2021): 02004. http://dx.doi.org/10.1051/e3sconf/202123302004.

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In recent years, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated nuclease (Cas) technology has become the most heated genome editing technique. Comparing to earlier genetic engineering methods, the CRISPR/Cas system is more advantageous due to its simple convenient design, high efficiency, cost-effectiveness, and the ability to perform multi-sites editing simultaneously. As the most effective gene editing tool, it utilizes a simple short RNA-guided mechanism to direct Cas-mediated DNA cleavage at the target genome locus and exploits the endogenous DNA r

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S., Manasa M. "CRISPR-Cas9 gene editing technology in human gene therapy: the new realm of medicine." International Journal of Advances in Medicine 9, no. 4 (2022): 513. http://dx.doi.org/10.18203/2349-3933.ijam20220796.

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Gene therapy has a huge clinical relevance in the present therapeutic world and is one of the many research fields of biology which received many benefits from the recent advancements of modern clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing technology. Researchers are on the way to make significant changes in the ways of treating genetic abnormalities. An increase in the number of approved clinical trials of CRISPR based gene therapy shows we are not too far from eliminating deadly diseases such as acquired immunodeficiency syndrome (AIDS), cancer and many

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Preece, Roland, and Christos Georgiadis. "Emerging CRISPR/Cas9 applications for T-cell gene editing." Emerging Topics in Life Sciences 3, no. 3 (2019): 261–75. http://dx.doi.org/10.1042/etls20180144.

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Abstract Gene editing tools are being rapidly developed, accelerating many areas of cell and gene therapy research. Each successive gene editing technology promises increased efficacy, improved specificity, reduced manufacturing cost and design complexity; all of which are currently epitomised by the clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein (Cas9) platform. Since its conceptualisation, CRISPR-based gene editing has been applied to existing methodologies and has further allowed the exploration of novel avenues of research. Implementation o

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Liu, Wenlou, Chunsheng Yang, Yanqun Liu, and Guan Jiang. "CRISPR/Cas9 System and its Research Progress in Gene Therapy." Anti-Cancer Agents in Medicinal Chemistry 19, no. 16 (2020): 1912–19. http://dx.doi.org/10.2174/1871520619666191014103711.

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Genome editing refers to changing the genome sequence of an organism by knockout, insertion, and site mutation, resulting in changes in the genetic information of the organism. The clustered regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated protein-9 nuclease (Cas9) system is a genome editing technique developed by the acquired immune system in the microbes, such as bacteria and archaebacteria, which targets and edits genome sequences according to the principle of complementary base pairing. This technique can be used to edit endogenous genomic DNA sequences in organi

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Salsman, Jayme, and Graham Dellaire. "Precision genome editing in the CRISPR era." Biochemistry and Cell Biology 95, no. 2 (2017): 187–201. http://dx.doi.org/10.1139/bcb-2016-0137.

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With the introduction of precision genome editing using CRISPR–Cas9 technology, we have entered a new era of genetic engineering and gene therapy. With RNA-guided endonucleases, such as Cas9, it is possible to engineer DNA double strand breaks (DSB) at specific genomic loci. DSB repair by the error-prone non-homologous end-joining (NHEJ) pathway can disrupt a target gene by generating insertions and deletions. Alternatively, Cas9-mediated DSBs can be repaired by homology-directed repair (HDR) using an homologous DNA repair template, thus allowing precise gene editing by incorporating genetic c

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10

Kanu, Gayathri A., Javad B. M. Parambath, Raed O. Abu Odeh, and Ahmed A. Mohamed. "Gold Nanoparticle-Mediated Gene Therapy." Cancers 14, no. 21 (2022): 5366. http://dx.doi.org/10.3390/cancers14215366.

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Gold nanoparticles (AuNPs) have gained increasing attention as novel drug-delivery nanostructures for the treatment of cancers, infections, inflammations, and other diseases and disorders. They are versatile in design, synthesis, modification, and functionalization. This has many advantages in terms of gene editing and gene silencing, and their application in genetic illnesses. The development of several techniques such as CRISPR/Cas9, TALEN, and ZFNs has raised hopes for the treatment of genetic abnormalities, although more focused experimentation is still needed. AuNPs, however, have been mu

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Khan, Sikandar Hayat. "Type-2 Diabetes and Gene Therapy: The Promise of CRISPR Gene Therapy in type-2 Diabetes Mellitus." Journal Of Obesity Management 1, no. 3 (2019): 1–5. http://dx.doi.org/10.14302/issn.2574-450x.jom-19-3001.

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Gene therapy has entered a new era with the dawn of CRISPR/Cas9 technology which though were always available in nature but rediscovered to tame into a real-tlife genome editing tool. With the modernization upsurge and changes in ways the “homo sapiens” survived on this planet from hunger to current era of exuberance has led to multiple metabolic issues like type-2 diabetes. Notwithstanding the rapid emergence of medication to suppress the hyperglycemia and insulin resistance associated with this menace, need has definitely emerge to find more personalized and curative dimensions to therapeuti

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Naso, Gaetano, and Anastasia Petrova. "CRISPR/Cas9 gene editing for genodermatoses: progress and perspectives." Emerging Topics in Life Sciences 3, no. 3 (2019): 313–26. http://dx.doi.org/10.1042/etls20180148.

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Abstract Genodermatoses constitute a clinically heterogeneous group of devastating genetic skin disorders. Currently, therapy options are largely limited to symptomatic treatments and although significant advances have been made in ex vivo gene therapy strategies, various limitations remain. However, the recent technical transformation of the genome editing field promises to overcome the hurdles associated with conventional gene addition approaches. In this review, we discuss the need for developing novel treatments and describe the current status of gene editing for genodermatoses, focusing o

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Koniali, Lola, Carsten W. Lederer, and Marina Kleanthous. "Therapy Development by Genome Editing of Hematopoietic Stem Cells." Cells 10, no. 6 (2021): 1492. http://dx.doi.org/10.3390/cells10061492.

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Accessibility of hematopoietic stem cells (HSCs) for the manipulation and repopulation of the blood and immune systems has placed them at the forefront of cell and gene therapy development. Recent advances in genome-editing tools, in particular for clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) and CRISPR/Cas-derived editing systems, have transformed the gene therapy landscape. Their versatility and the ability to edit genomic sequences and facilitate gene disruption, correction or insertion, have broadened the spectrum of potential gene ther

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Bonillo, Mario, Julia Pfromm, and M. Dominik Fischer. "Challenges to Gene Editing Approaches in the Retina." Klinische Monatsblätter für Augenheilkunde 239, no. 03 (2022): 275–83. http://dx.doi.org/10.1055/a-1757-9810.

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AbstractRetinal gene therapy has recently been at the cutting edge of clinical development in the diverse field of genetic therapies. The retina is an attractive target for genetic therapies such as gene editing due to the distinctive anatomical and immunological features of the eye, known as immune privilege, so that inherited retinal diseases (IRDs) have been studied in several clinical studies. Thus, rapid strides are being made toward developing targeted treatments for IRDs. Gene editing in the retina faces a group of heterogenous challenges, including editing efficiencies, off-target effe

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Rosenblum, Daniel, Anna Gutkin, Ranit Kedmi, et al. "CRISPR-Cas9 genome editing using targeted lipid nanoparticles for cancer therapy." Science Advances 6, no. 47 (2020): eabc9450. http://dx.doi.org/10.1126/sciadv.abc9450.

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Harnessing CRISPR-Cas9 technology for cancer therapeutics has been hampered by low editing efficiency in tumors and potential toxicity of existing delivery systems. Here, we describe a safe and efficient lipid nanoparticle (LNP) for the delivery of Cas9 mRNA and sgRNAs that use a novel amino-ionizable lipid. A single intracerebral injection of CRISPR-LNPs against PLK1 (sgPLK1-cLNPs) into aggressive orthotopic glioblastoma enabled up to ~70% gene editing in vivo, which caused tumor cell apoptosis, inhibited tumor growth by 50%, and improved survival by 30%. To reach disseminated tumors, cLNPs w

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García-Fernández, Alba, Gema Vivo-Llorca, Mónica Sancho, et al. "Nanodevices for the Efficient Codelivery of CRISPR-Cas9 Editing Machinery and an Entrapped Cargo: A Proposal for Dual Anti-Inflammatory Therapy." Pharmaceutics 14, no. 7 (2022): 1495. http://dx.doi.org/10.3390/pharmaceutics14071495.

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In this article, we report one of the few examples of nanoparticles capable of simultaneously delivering CRISPR-Cas9 gene-editing machinery and releasing drugs for one-shot treatments. Considering the complexity of inflammation in diseases, the synergistic effect of nanoparticles for gene-editing/drug therapy is evaluated in an in vitro inflammatory model as proof of concept. Mesoporous silica nanoparticles (MSNs), able to deliver the CRISPR/Cas9 machinery to edit gasdermin D (GSDMD), a key protein involved in inflammatory cell death, and the anti-inflammatory drug VX-765 (GSDMD45CRISPR-VX-MSN

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Fang, Ton, Goun Je, Peter Pacut, Kiandokht Keyhanian, Jeff Gao, and Mehdi Ghasemi. "Gene Therapy in Amyotrophic Lateral Sclerosis." Cells 11, no. 13 (2022): 2066. http://dx.doi.org/10.3390/cells11132066.

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Since the discovery of Cu/Zn superoxide dismutase (SOD1) gene mutation, in 1993, as the first genetic abnormality in amyotrophic lateral sclerosis (ALS), over 50 genes have been identified as either cause or modifier in ALS and ALS/frontotemporal dementia (FTD) spectrum disease. Mutations in C9orf72, SOD1, TAR DNA binding protein 43 (TARDBP), and fused in sarcoma (FUS) genes are the four most common ones. During the last three decades, tremendous effort has been made worldwide to reveal biological pathways underlying the pathogenesis of these gene mutations in ALS/FTD. Accordingly, targeting e

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Ren, Duohao, Sylvain Fisson, Deniz Dalkara, and Divya Ail. "Immune Responses to Gene Editing by Viral and Non-Viral Delivery Vectors Used in Retinal Gene Therapy." Pharmaceutics 14, no. 9 (2022): 1973. http://dx.doi.org/10.3390/pharmaceutics14091973.

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Inherited retinal diseases (IRDs) are a leading cause of blindness in industrialized countries, and gene therapy is quickly becoming a viable option to treat this group of diseases. Gene replacement using a viral vector has been successfully applied and advanced to commercial use for a rare group of diseases. This, and the advances in gene editing, are paving the way for the emergence of a new generation of therapies that use CRISPR–Cas9 to edit mutated genes in situ. These CRISPR-based agents can be delivered to the retina as transgenes in a viral vector, unpackaged transgenes or as proteins

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Psatha, Nikoletta, Kiriaki Paschoudi, Anastasia Papadopoulou, and Evangelia Yannaki. "In Vivo Hematopoietic Stem Cell Genome Editing: Perspectives and Limitations." Genes 13, no. 12 (2022): 2222. http://dx.doi.org/10.3390/genes13122222.

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The tremendous evolution of genome-editing tools in the last two decades has provided innovative and effective approaches for gene therapy of congenital and acquired diseases. Zinc-finger nucleases (ZFNs), transcription activator- like effector nucleases (TALENs) and CRISPR-Cas9 have been already applied by ex vivo hematopoietic stem cell (HSC) gene therapy in genetic diseases (i.e., Hemoglobinopathies, Fanconi anemia and hereditary Immunodeficiencies) as well as infectious diseases (i.e., HIV), and the recent development of CRISPR-Cas9-based systems using base and prime editors as well as epi

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Kesavan, Gokul. "Emerging Gene Editing Therapies for Blood Disorders." Biotechnology Kiosk 3, no. 7 (2021): 3–16. http://dx.doi.org/10.37756/bk.21.3.7.1.

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Hemoglobinopathies like sickle cell disease (SCD) and beta thalassemia are the most common monogenic disorders and the number of children born every year with such conditions is predicted to reach about 400,000 by the year 2050. Transplantation of hematopoietic stem cells remains the gold standard of treatment but finding a Human Leukocyte Antigen (HLA)-matched donor is a major bottle neck when large numbers of patients need to be treated. Recent advances in gene editing tools, like Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR), show immense potential to transform the gene

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Sun, Jinyu, Jianchu Wang, Donghui Zheng, and Xiaorong Hu. "Advances in therapeutic application of CRISPR-Cas9." Briefings in Functional Genomics 19, no. 3 (2019): 164–74. http://dx.doi.org/10.1093/bfgp/elz031.

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Abstract Clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) is one of the most versatile and efficient gene editing technologies, which is derived from adaptive immune strategies for bacteria and archaea. With the remarkable development of programmable nuclease-based genome engineering these years, CRISPR-Cas9 system has developed quickly in recent 5 years and has been widely applied in countless areas, including genome editing, gene function investigation and gene therapy both in vitro and in vivo. In this paper, we briefly introduce the mechanisms

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Zhao, Lan, Jian Huang, Yunshan Fan, et al. "Exploration of CRISPR/Cas9-based gene editing as therapy for osteoarthritis." Annals of the Rheumatic Diseases 78, no. 5 (2019): 676–82. http://dx.doi.org/10.1136/annrheumdis-2018-214724.

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ObjectivesOsteoarthritis (OA) is a painful and debilitating disease and it is associated with aberrant upregulation of multiple factors, including matrix metalloproteinase 13 (MMP13), interleukin-1β (IL-1β) and nerve growth factor (NGF). In this study, we aimed to use the CRISPR/Cas9 technology, a highly efficient gene-editing tool, to study whether the ablation of OA-associated genes has OA-modifying effects.MethodsWe performed intra-articular injection of adeno-associated virus, which expressed CRISPR/Cas9 components to target each of the genes encoding MMP13, IL-1β and NGF, in a surgically

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Kochergin-Nikitsky, K. S., E. V. Zaklyazminskaya, A. V. Lavrov, and S. A. Smirnikhina. "Cardiomyopathies associated with the DES gene mutations: molecular pathogenesis and gene therapy approaches." Almanac of Clinical Medicine 47, no. 7 (2019): 603–13. http://dx.doi.org/10.18786/2072-0505-2019-47-025.

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Cardiomyopathy (CMP) is a common group of cardiovascular disorders. Genetic (primary) cardiomyopathies are related to abnormalities in more than 100 genes, including the DES gene encoding desmin protein. Desmin is an essential member of the intermediate filaments, ensuring the structural and functional integrity of myocytes. Mutations in the DES gene result in desmin-related cardiomyopathy with progressive course and poor prognosis. By now, specific therapy for cardiomyopathy has not been developed. Existing conservative and surgical treatment modalities target the rate of heart failure progre

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Meiliana, Anna, Nurrani Mustika Dewi, and Andi Wijaya. "Genome Editing with Crispr-Cas9 Systems: Basic Research and Clinical Applications." Indonesian Biomedical Journal 9, no. 1 (2017): 1. http://dx.doi.org/10.18585/inabj.v9i1.272.

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BACKGROUND: Recently established genome editing technologies will open new avenues for biological research and development. Human genome editing is a powerful tool which offers great scientific and therapeutic potential.CONTENT: Genome editing using the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPRassociated protein 9 (Cas9) technology is revolutionizing the gene function studies and possibly will give rise to an entirely new degree of therapeutics for a large range of diseases. Prompt advances in the CRISPR/Cas9 technology, as well as delivery modalities for gene t

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Teng, Man, Yongxiu Yao, Venugopal Nair, and Jun Luo. "Latest Advances of Virology Research Using CRISPR/Cas9-Based Gene-Editing Technology and Its Application to Vaccine Development." Viruses 13, no. 5 (2021): 779. http://dx.doi.org/10.3390/v13050779.

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In recent years, the CRISPR/Cas9-based gene-editing techniques have been well developed and applied widely in several aspects of research in the biological sciences, in many species, including humans, animals, plants, and even in viruses. Modification of the viral genome is crucial for revealing gene function, virus pathogenesis, gene therapy, genetic engineering, and vaccine development. Herein, we have provided a brief review of the different technologies for the modification of the viral genomes. Particularly, we have focused on the recently developed CRISPR/Cas9-based gene-editing system,

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Shaikh, Sadiya Bi, and Yashodhar Prabhakar Bhandary. "CRISPR/Cas9 Genome Editing Tool: A Promising Tool for Therapeutic Applications on Respiratory Diseases." Current Gene Therapy 20, no. 5 (2020): 333–46. http://dx.doi.org/10.2174/1566523220666201012145731.

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Respiratory diseases are one of the prime topics of concern in the current era due to improper diagnostics tools. Gene-editing therapy, like Clustered regularly interspaced palindromic repeats- associated nuclease 9 (CRISPR/Cas9), is gaining popularity in pulmonary research, opening up doors to invaluable insights on underlying mechanisms. CRISPR/Cas9 can be considered as a potential gene-editing tool with a scientific community that is helping in the advancement of knowledge in respiratory health and therapy. As an appealing therapeutic tool, we hereby explore the advanced research on the app

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Gamage, Udani, Kesari Warnakulasuriya, Sonali Hansika та Gayathri N. Silva. "CRISPR Gene Therapy: A Promising One-Time Therapeutic Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia". Thalassemia Reports 13, № 1 (2023): 51–69. http://dx.doi.org/10.3390/thalassrep13010006.

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β-Thalassemia is an inherited hematological disorder that results from genetic changes in the β-globin gene, leading to the reduced or absent synthesis of β-globin. For several decades, the only curative treatment option for β-thalassemia has been allogeneic hematopoietic cell transplantation (allo-HCT). Nonetheless, rapid progress in genome modification technologies holds great potential for treating this disease and will soon change the current standard of care for β-thalassemia. For instance, the emergence of the CRISPR/Cas9 genome editing platform has opened the door for precision gene edi

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Zhang, Zhihao, Wei Hou, and Shuliang Chen. "Updates on CRISPR-based gene editing in HIV-1/AIDS therapy." Virologica Sinica 37, no. 1 (2022): 1–10. http://dx.doi.org/10.1016/j.virs.2022.01.017.

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Nakamura, Watanabe, Ando, Ishihara, and Sato. "Transplacental Gene Delivery (TPGD) as a Noninvasive Tool for Fetal Gene Manipulation in Mice." International Journal of Molecular Sciences 20, no. 23 (2019): 5926. http://dx.doi.org/10.3390/ijms20235926.

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Transplacental gene delivery (TPGD) is a technique for delivering nucleic acids to fetal tissues via tail-vein injections in pregnant mice. After transplacental transport, administered nucleic acids enter fetal circulation and are distributed among fetal tissues. TPGD was established in 1995 by Tsukamoto et al., and its mechanisms, and potential applications have been further characterized since. Recently, discoveries of sequence specific nucleases, such as zinc-finger nuclease (ZFN), transcription activator-like effector nucleases (TALEN), and clustered regularly interspaced short palindromic

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Nie, Dengyun, Ting Guo, Miao Yue, et al. "Research Progress on Nanoparticles-Based CRISPR/Cas9 System for Targeted Therapy of Tumors." Biomolecules 12, no. 9 (2022): 1239. http://dx.doi.org/10.3390/biom12091239.

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Cancer is a genetic mutation disease that seriously endangers the health and life of all human beings. As one of the most amazing academic achievements in the past decade, CRISPR/Cas9 technology has been sought after by many researchers due to its powerful gene editing capability. CRISPR/Cas9 technology shows great potential in oncology, and has become one of the most promising technologies for cancer genome-editing therapeutics. However, its efficiency and the safety issues of in vivo gene editing severely limit its widespread application. Therefore, developing a suitable delivery method for

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Yu, Jiaying, Xi Xiang, Jinrong Huang, et al. "Haplotyping by CRISPR-mediated DNA circularization (CRISPR-hapC) broadens allele-specific gene editing." Nucleic Acids Research 48, no. 5 (2020): e25-e25. http://dx.doi.org/10.1093/nar/gkz1233.

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Abstract Allele-specific protospacer adjacent motif (asPAM)-positioning SNPs and CRISPRs are valuable resources for gene therapy of dominant disorders. However, one technical hurdle is to identify the haplotype comprising the disease-causing allele and the distal asPAM SNPs. Here, we describe a novel CRISPR-based method (CRISPR-hapC) for haplotyping. Based on the generation (with a pair of CRISPRs) of extrachromosomal circular DNA in cells, the CRISPR-hapC can map haplotypes from a few hundred bases to over 200 Mb. To streamline and demonstrate the applicability of the CRISPR-hapC and asPAM CR

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Godbout, Kelly, and Jacques P. Tremblay. "Prime Editing for Human Gene Therapy: Where Are We Now?" Cells 12, no. 4 (2023): 536. http://dx.doi.org/10.3390/cells12040536.

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Gene therapy holds tremendous potential in the treatment of inherited diseases. Unlike traditional medicines, which only treat the symptoms, gene therapy has the potential to cure the disease by addressing the root of the problem: genetic mutations. The discovery of CRISPR/Cas9 in 2012 paved the way for the development of those therapies. Improvement of this system led to the recent development of an outstanding technology called prime editing. This system can introduce targeted insertions, deletions, and all 12 possible base-to-base conversions in the human genome. Since the first publication

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Balon, Katarzyna, Adam Sheriff, Joanna Jacków, and Łukasz Łaczmański. "Targeting Cancer with CRISPR/Cas9-Based Therapy." International Journal of Molecular Sciences 23, no. 1 (2022): 573. http://dx.doi.org/10.3390/ijms23010573.

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Cancer is a devastating condition characterised by the uncontrolled division of cells with many forms remaining resistant to current treatment. A hallmark of cancer is the gradual accumulation of somatic mutations which drive tumorigenesis in cancerous cells, creating a mutation landscape distinctive to a cancer type, an individual patient or even a single tumour lesion. Gene editing with CRISPR/Cas9-based tools now enables the precise and permanent targeting of mutations and offers an opportunity to harness this technology to target oncogenic mutations. However, the development of safe and ef

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Lebek, Simon, Francesco Chemello, Xurde M. Caravia та ін. "Ablation of CaMKIIδ oxidation by CRISPR-Cas9 base editing as a therapy for cardiac disease". Science 379, № 6628 (2023): 179–85. http://dx.doi.org/10.1126/science.ade1105.

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CRISPR-Cas9 gene editing is emerging as a prospective therapy for genomic mutations. However, current editing approaches are directed primarily toward relatively small cohorts of patients with specific mutations. Here, we describe a cardioprotective strategy potentially applicable to a broad range of patients with heart disease. We used base editing to ablate the oxidative activation sites of CaMKIIδ, a primary driver of cardiac disease. We show in cardiomyocytes derived from human induced pluripotent stem cells that editing the CaMKIIδ gene to eliminate oxidation-sensitive methionine residues

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Benati, Daniela, Amy Leung, Pedro Perdigao, Vasileios Toulis, Jacqueline van der Spuy, and Alessandra Recchia. "Induced Pluripotent Stem Cells and Genome-Editing Tools in Determining Gene Function and Therapy for Inherited Retinal Disorders." International Journal of Molecular Sciences 23, no. 23 (2022): 15276. http://dx.doi.org/10.3390/ijms232315276.

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Inherited retinal disorders (IRDs) affect millions of people worldwide and are a major cause of irreversible blindness. Therapies based on drugs, gene augmentation or transplantation approaches have been widely investigated and proposed. Among gene therapies for retinal degenerative diseases, the fast-evolving genome-editing CRISPR/Cas technology has emerged as a new potential treatment. The CRISPR/Cas system has been developed as a powerful genome-editing tool in ophthalmic studies and has been applied not only to gain proof of principle for gene therapies in vivo, but has also been extensive

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Lyu, Pin, Luxi Wang, and Baisong Lu. "Virus-Like Particle Mediated CRISPR/Cas9 Delivery for Efficient and Safe Genome Editing." Life 10, no. 12 (2020): 366. http://dx.doi.org/10.3390/life10120366.

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The discovery of designer nucleases has made genome editing much more efficient than before. The designer nucleases have been widely used for mechanistic studies, animal model generation and gene therapy development. However, potential off-targets and host immune responses are issues still need to be addressed for in vivo uses, especially clinical applications. Short term expression of the designer nucleases is necessary to reduce both risks. Currently, various delivery methods are being developed for transient expression of designer nucleases including Zinc Finger Nuclease (ZNF), Transcriptio

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Zhou, Jun, Zhuoying Ren, Jie Xu, Jifeng Zhang, and Y. Eugene Chen. "Gene editing therapy ready for cardiovascular diseases: opportunities, challenges, and perspectives." Medical Review 1, no. 1 (2021): 6–9. http://dx.doi.org/10.1515/mr-2021-0010.

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Abstract Gene editing nucleases (GENs), represented by CRISPR/Cas9, have become major tools in biomedical research and offer potential cures for many human diseases. Gene editing therapy (GETx) studies in animal models targeting genes such as proprotein convertase subtilisin/kexin type 9 (PCSK9), apolipoprotein C3 (APOC3), angiopoietin Like 3 (ANGPTL3) and inducible degrader of the low-density lipoprotein receptor (IDOL) have demonstrated the benefits and advantages of GETx in managing atherosclerosis. Here we present our views on this brand new therapeutic option for cardiovascular diseases (

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Santos, Renato, and Olga Amaral. "Advances in Sphingolipidoses: CRISPR-Cas9 Editing as an Option for Modelling and Therapy." International Journal of Molecular Sciences 20, no. 23 (2019): 5897. http://dx.doi.org/10.3390/ijms20235897.

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Sphingolipidoses are inherited genetic diseases characterized by the accumulation of glycosphingolipids. Sphingolipidoses (SP), which usually involve the loss of sphingolipid hydrolase function, are of lysosomal origin, and represent an important group of rare diseases among lysosomal storage disorders. Initial treatments consisted of enzyme replacement therapy, but, in recent decades, various therapeutic approaches have been developed. However, these commonly used treatments for SP fail to be fully effective and do not penetrate the blood–brain barrier. New approaches, such as genome editing,

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Xiu, Kemao, Laura Saunders, Luan Wen, et al. "Delivery of CRISPR/Cas9 Plasmid DNA by Hyperbranched Polymeric Nanoparticles Enables Efficient Gene Editing." Cells 12, no. 1 (2022): 156. http://dx.doi.org/10.3390/cells12010156.

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Gene editing nucleases such as CRISPR/Cas9 have enabled efficient and precise gene editing in vitro and hold promise of eventually achieving in vivo gene editing based therapy. However, a major challenge for their use is the lack of a safe and effective virus-free system to deliver gene editing nuclease elements. Polymers are a promising class of delivery vehicle due to their higher safety compared to currently used viral vectors, but polymers suffer from lower transfection efficiency. Polymeric vectors have been used for small nucleotide delivery but have yet to be used successfully with plas

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Padmaswari, Made Harumi, Shilpi Agrawal, Mary S. Jia, et al. "Delivery challenges for CRISPR—Cas9 genome editing for Duchenne muscular dystrophy." Biophysics Reviews 4, no. 1 (2023): 011307. http://dx.doi.org/10.1063/5.0131452.

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Duchene muscular dystrophy (DMD) is an X-linked neuromuscular disorder that affects about one in every 5000 live male births. DMD is caused by mutations in the gene that codes for dystrophin, which is required for muscle membrane stabilization. The loss of functional dystrophin causes muscle degradation that leads to weakness, loss of ambulation, cardiac and respiratory complications, and eventually, premature death. Therapies to treat DMD have advanced in the past decade, with treatments in clinical trials and four exon-skipping drugs receiving conditional Food and Drug Administration approva

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Yan, Biying, and Yaxuan Liang. "New Therapeutics for Extracellular Vesicles: Delivering CRISPR for Cancer Treatment." International Journal of Molecular Sciences 23, no. 24 (2022): 15758. http://dx.doi.org/10.3390/ijms232415758.

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Cancers are defined by genetic defects, which underlines the prospect of using gene therapy in patient care. During the past decade, CRISPR technology has rapidly evolved into a powerful gene editing tool with high fidelity and precision. However, one of the impediments slowing down the clinical translation of CRISPR-based gene therapy concerns the lack of ideal delivery vectors. Extracellular vesicles (EVs) are nano-sized membrane sacs naturally released from nearly all types of cells. Although EVs are secreted for bio-information conveyance among cells or tissues, they have been recognized a

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Rabaan, Ali A., Hajir AlSaihati, Rehab Bukhamsin, et al. "Application of CRISPR/Cas9 Technology in Cancer Treatment: A Future Direction." Current Oncology 30, no. 2 (2023): 1954–76. http://dx.doi.org/10.3390/curroncol30020152.

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Gene editing, especially with clustered regularly interspaced short palindromic repeats associated protein 9 (CRISPR-Cas9), has advanced gene function science. Gene editing’s rapid advancement has increased its medical/clinical value. Due to its great specificity and efficiency, CRISPR/Cas9 can accurately and swiftly screen the whole genome. This simplifies disease-specific gene therapy. To study tumor origins, development, and metastasis, CRISPR/Cas9 can change genomes. In recent years, tumor treatment research has increasingly employed this method. CRISPR/Cas9 can treat cancer by removing ge

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Hou, Yujuan, Guillermo Ureña-Bailén, Tahereh Mohammadian Gol, et al. "Challenges in Gene Therapy for Somatic Reverted Mosaicism in X-Linked Combined Immunodeficiency by CRISPR/Cas9 and Prime Editing." Genes 13, no. 12 (2022): 2348. http://dx.doi.org/10.3390/genes13122348.

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X-linked severe combined immunodeficiency (X-SCID) is a primary immunodeficiency that is caused by mutations in the interleukin-2 receptor gamma (IL2RG) gene. Some patients present atypical X-SCID with mild clinical symptoms due to somatic revertant mosaicism. CRISPR/Cas9 and prime editing are two advanced genome editing tools that paved the way for treating immune deficiency diseases. Prime editing overcomes the limitations of the CRISPR/Cas9 system, as it does not need to induce double-strand breaks (DSBs) or exogenous donor DNA templates to modify the genome. Here, we applied CRISPR/Cas9 wi

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Chen, Guofang, Tingyi Wei, Hui Yang, Guoling Li, and Haisen Li. "CRISPR-Based Therapeutic Gene Editing for Duchenne Muscular Dystrophy: Advances, Challenges and Perspectives." Cells 11, no. 19 (2022): 2964. http://dx.doi.org/10.3390/cells11192964.

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Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease arising from loss-of-function mutations in the dystrophin gene and characterized by progressive muscle degeneration, respiratory insufficiency, cardiac failure, and premature death by the age of thirty. Albeit DMD is one of the most common types of fatal genetic diseases, there is no curative treatment for this devastating disorder. In recent years, gene editing via the clustered regularly interspaced short palindromic repeats (CRISPR) system has paved a new path toward correcting pathological mutations at the genetic source,

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Bischoff, Nadja, Sandra Wimberger, Marcello Maresca, and Cord Brakebusch. "Improving Precise CRISPR Genome Editing by Small Molecules: Is there a Magic Potion?" Cells 9, no. 5 (2020): 1318. http://dx.doi.org/10.3390/cells9051318.

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Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) genome editing has become a standard method in molecular biology, for the establishment of genetically modified cellular and animal models, for the identification and validation of drug targets in animals, and is heavily tested for use in gene therapy of humans. While the efficiency of CRISPR mediated gene targeting is much higher than of classical targeted mutagenesis, the efficiency of CRISPR genome editing to introduce defined changes into the genome is still low. Overcoming this problem will have a great impact on the use o

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Huang, Yong, Meiqi Shang, Tingting Liu, and Kejian Wang. "High-throughput methods for genome editing: the more the better." Plant Physiology 188, no. 4 (2022): 1731–45. http://dx.doi.org/10.1093/plphys/kiac017.

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Abstract During the last decade, targeted genome-editing technologies, especially clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein (Cas) technologies, have permitted efficient targeting of genomes, thereby modifying these genomes to offer tremendous opportunities for deciphering gene function and engineering beneficial traits in many biological systems. As a powerful genome-editing tool, the CRISPR/Cas systems, combined with the development of next-generation sequencing and many other high-throughput techniques, have thus been quickly developed into a

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Hussein, Mouraya, Mariano A. Molina, Ben Berkhout, and Elena Herrera-Carrillo. "A CRISPR-Cas Cure for HIV/AIDS." International Journal of Molecular Sciences 24, no. 2 (2023): 1563. http://dx.doi.org/10.3390/ijms24021563.

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Human immunodeficiency virus (HIV) infections and HIV-induced acquired immunodeficiency syndrome (AIDS) continue to represent a global health burden. There is currently no effective vaccine, nor any cure, for HIV infections; existing antiretroviral therapy can suppress viral replication, but only as long as antiviral drugs are taken. HIV infects cells of the host immune system, and it can establish a long-lived viral reservoir, which can be targeted and edited through gene therapy. Gene editing platforms based on the clustered regularly interspaced palindromic repeat-Cas system (CRISPR-Cas) ha

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Omichi, Ryotaro, Seiji B. Shibata, Cynthia C. Morton, and Richard J. H. Smith. "Gene therapy for hearing loss." Human Molecular Genetics 28, R1 (2019): R65—R79. http://dx.doi.org/10.1093/hmg/ddz129.

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Abstract Sensorineural hearing loss (SNHL) is the most common sensory disorder. Its underlying etiologies include a broad spectrum of genetic and environmental factors that can lead to hearing loss that is congenital or late onset, stable or progressive, drug related, noise induced, age related, traumatic or post-infectious. Habilitation options typically focus on amplification using wearable or implantable devices; however exciting new gene-therapy-based strategies to restore and prevent SNHL are actively under investigation. Recent proof-of-principle studies demonstrate the potential therape

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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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Nasrallah, Ali, Eric Sulpice, Farah Kobaisi, Xavier Gidrol, and Walid Rachidi. "CRISPR-Cas9 Technology for the Creation of Biological Avatars Capable of Modeling and Treating Pathologies: From Discovery to the Latest Improvements." Cells 11, no. 22 (2022): 3615. http://dx.doi.org/10.3390/cells11223615.

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This is a spectacular moment for genetics to evolve in genome editing, which encompasses the precise alteration of the cellular DNA sequences within various species. One of the most fascinating genome-editing technologies currently available is Clustered Regularly Interspaced Palindromic Repeats (CRISPR) and its associated protein 9 (CRISPR-Cas9), which have integrated deeply into the research field within a short period due to its effectiveness. It became a standard tool utilized in a broad spectrum of biological and therapeutic applications. Furthermore, reliable disease models are required

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