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Journal articles on the topic 'Correction génique (CRISPR/Cas9)'

Author: Grafiati

Published: 7 July 2024

Last updated: 31 July 2025

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1

Jordan, Bertrand. "CRISPR-Cas9, une nouvelle donne pour la thérapie génique." médecine/sciences 31, no. 11 (2015): 1035–38. http://dx.doi.org/10.1051/medsci/20153111018.

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Cheng, Hao, Feng Zhang, and Yang Ding. "CRISPR/Cas9 Delivery System Engineering for Genome Editing in Therapeutic Applications." Pharmaceutics 13, no. 10 (2021): 1649. http://dx.doi.org/10.3390/pharmaceutics13101649.

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The clustered regularly interspaced short palindromic repeats (CRISPR)/associated protein 9 (CRISPR/Cas9) systems have emerged as a robust and versatile genome editing platform for gene correction, transcriptional regulation, disease modeling, and nucleic acids imaging. However, the insufficient transfection and off-target risks have seriously hampered the potential biomedical applications of CRISPR/Cas9 technology. Herein, we review the recent progress towards CRISPR/Cas9 system delivery based on viral and non-viral vectors. We summarize the CRISPR/Cas9-inspired clinical trials and analyze th

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Malki, Hugo, Juliette Pulman, and Deniz Dalkara. "Édition génique par transfert de complexes ribonucléoprotéiques de CRISPR/Cas9 dans la rétine." médecine/sciences 41, no. 6-7 (2025): 540–42. https://doi.org/10.1051/medsci/2025091.

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Abdelmoula, B., and N. Bouayed Abdelmoula. "CRISPR/Cas9 genome editing clinical trials for neurodevelopmental disorders." European Psychiatry 67, S1 (2024): S584—S585. http://dx.doi.org/10.1192/j.eurpsy.2024.1216.

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IntroductionRecently, the new therapeutic approach based on genome editing using the CRISPR/Cas9 system has been applied to treat cancer and other monogenetic disorders. CRISPR/Cas9 allows specific correction of the altered gene without affecting the rest of the genome.ObjectivesThe aim of this study was to report the current CRISPR/Cas9 genome editing clinical trials in neurodevelopmental and mental disorders.Methods We conducted a search via the ClinicalTrials platform to describe clinical trials that have been conducted using the CRISPR/Cas9 genome-editing tool in neurodevelopmental disorde

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Walther, Johanna, Danny Wilbie, Vincent S. J. Tissingh, et al. "Impact of Formulation Conditions on Lipid Nanoparticle Characteristics and Functional Delivery of CRISPR RNP for Gene Knock-Out and Correction." Pharmaceutics 14, no. 1 (2022): 213. http://dx.doi.org/10.3390/pharmaceutics14010213.

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The CRISPR-Cas9 system is an emerging therapeutic tool with the potential to correct diverse genetic disorders. However, for gene therapy applications, an efficient delivery vehicle is required, capable of delivering the CRISPR-Cas9 components into the cytosol of the intended target cell population. In this study, we optimized the formulation conditions of lipid nanoparticles (LNP) for delivery of ready-made CRISPR-Cas9 ribonucleic protein (RNP). The buffer composition during complexation and relative DOTAP concentrations were varied for LNP encapsulating in-house produced Cas9 RNP alone or Ca

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Yun, Yeomin, and Yoon Ha. "CRISPR/Cas9-Mediated Gene Correction to Understand ALS." International Journal of Molecular Sciences 21, no. 11 (2020): 3801. http://dx.doi.org/10.3390/ijms21113801.

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Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease caused by the death of motor neurons in the spinal cord and brainstem. ALS has a diverse genetic origin; at least 20 genes have been shown to be related to ALS. Most familial and sporadic cases of ALS are caused by variants of the SOD1, C9orf72, FUS, and TARDBP genes. Genome editing using clustered regularly interspaced short palindromic repeats/CRISPR-associated system 9 (CRISPR/Cas9) can provide insights into the underlying genetics and pathophysiology of ALS. By correcting common mutations associated with ALS in animal model

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Men, Ke, Xingmei Duan, Zhiyao He, Yang Yang, Shaohua Yao, and Yuquan Wei. "CRISPR/Cas9-mediated correction of human genetic disease." Science China Life Sciences 60, no. 5 (2017): 447–57. http://dx.doi.org/10.1007/s11427-017-9032-4.

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Hainzl, S., P. Peking, T. Kocher, et al. "185 CRISPR/Cas9 mediated gene correction of COL7A1." Journal of Investigative Dermatology 137, no. 10 (2017): S224. http://dx.doi.org/10.1016/j.jid.2017.07.182.

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Hanafy, Amira Sayed, Susanne Schoch, and Alf Lamprecht. "CRISPR/Cas9 Delivery Potentials in Alzheimer’s Disease Management: A Mini Review." Pharmaceutics 12, no. 9 (2020): 801. http://dx.doi.org/10.3390/pharmaceutics12090801.

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Alzheimer’s disease (AD) is the most common dementia disorder. While genetic mutations account for only 1% of AD cases, sporadic AD resulting from a combination of genetic and risk factors constitutes >90% of the cases. Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-associated protein (Cas9) is an impactful gene editing tool which identifies a targeted gene sequence, creating a double-stranded break followed by gene inactivation or correction. Although CRISPR/Cas9 can be utilized to irreversibly inactivate or correct faulty genes in AD, a safe and effective delivery syst

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Haq, Ehsan Ul, Haseeb Khaliq, Ayesha Muddasser, et al. "EVALUATION OF CRISPR/CAS9 GENOME-EDITING SYSTEM IN HUMAN STEM CELLS HSCS: THERAPEUTICS AND DIAGNOSTICS PROSPECTS." Insights-Journal of Health and Rehabilitation 3, no. 3 (Health & Allied) (2025): 262–71. https://doi.org/10.71000/ce0xek27.

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Background: CRISPR/Cas9 genome-editing technology has revolutionized human stem cell (HSC) research, offering novel therapeutic and diagnostic applications. HSCs play a crucial role in regenerative medicine and genetic therapies due to their ability to self-renew and differentiate into various blood cell lineages. The precise genome-editing capability of CRISPR/Cas9 allows for targeted gene modifications, enabling the correction of inherited disorders, disease modeling and the discovery of novel biomarkers. Despite significant advancements, challenges such as off-target effects, delivery effic

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Jo, Dong Hyun, Dong Woo Song, Chang Sik Cho, et al. "CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis." Science Advances 5, no. 10 (2019): eaax1210. http://dx.doi.org/10.1126/sciadv.aax1210.

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Leber congenital amaurosis (LCA), one of the leading causes of childhood-onset blindness, is caused by autosomal recessive mutations in several genes including RPE65. In this study, we performed CRISPR-Cas9–mediated therapeutic correction of a disease-associated nonsense mutation in Rpe65 in rd12 mice, a model of human LCA. Subretinal injection of adeno-associated virus carrying CRISPR-Cas9 and donor DNA resulted in >1% homology-directed repair and ~1.6% deletion of the pathogenic stop codon in Rpe65 in retinal pigment epithelial tissues of rd12 mice. The a- and b-waves of electroretinogram

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Atmanli, Ayhan, Andreas C. Chai, Miao Cui, et al. "Cardiac Myoediting Attenuates Cardiac Abnormalities in Human and Mouse Models of Duchenne Muscular Dystrophy." Circulation Research 129, no. 6 (2021): 602–16. http://dx.doi.org/10.1161/circresaha.121.319579.

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Rationale: Absence of dystrophin in Duchenne muscular dystrophy (DMD) results in the degeneration of skeletal and cardiac muscles. Owing to advances in respiratory management of patients with DMD, cardiomyopathy has become a significant aspect of the disease. While CRISPR/Cas9 genome editing technology holds great potential as a novel therapeutic avenue for DMD, little is known about the potential of DMD correction using CRISPR/Cas9 technology to mitigate cardiac abnormalities in DMD. Objective: To define the effects of CRISPR/Cas9 genome editing on structural, functional, and transcriptional

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Luo, Yumei, Detu Zhu, Zhizhuo Zhang, Yaoyong Chen, and Xiaofang Sun. "Integrative Analysis of CRISPR/Cas9 Target Sites in the HumanHBBGene." BioMed Research International 2015 (2015): 1–9. http://dx.doi.org/10.1155/2015/514709.

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Recently, the clustered regularly interspaced short palindromic repeats (CRISPR) system has emerged as a powerful customizable artificial nuclease to facilitate precise genetic correction for tissue regeneration and isogenic disease modeling. However, previous studies reported substantial off-target activities of CRISPR system in human cells, and the enormous putative off-target sites are labor-intensive to be validated experimentally, thus motivating bioinformatics methods for rational design of CRISPR system and prediction of its potential off-target effects. Here, we describe an integrative

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Jinka, Chaitra. "CRISPR-Cas9 gene editing and human diseases." Bioinformation 18, no. 11 (2022): 1081–86. http://dx.doi.org/10.6026/973206300181081.

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CRISPR/Cas-9 mediated genome editing has recently emerged as a potential and innovative technology in therapeutic development and biomedical research. Several recent studies have been performed to understand gene modification techniques in obtaining effective ex vivo results. Generally, the disease targets for gene correction will be in specific organs, so understanding the complete potential of genomic treatment methods is essential. From such a perspective, the present review revealed the significant importance of the CRISPR/ Cas9 delivery system. Both the promising gene-editing delivery sys

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Pöhler, Michael, Sarah Guttmann, Oksana Nadzemova, et al. "CRISPR/Cas9-mediated correction of mutated copper transporter ATP7B." PLOS ONE 15, no. 9 (2020): e0239411. http://dx.doi.org/10.1371/journal.pone.0239411.

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Xia, Emily, Rongqi Duan, Fushan Shi, Kyle E. Seigel, Hartmut Grasemann, and Jim Hu. "Overcoming the Undesirable CRISPR-Cas9 Expression in Gene Correction." Molecular Therapy - Nucleic Acids 13 (December 2018): 699–709. http://dx.doi.org/10.1016/j.omtn.2018.10.015.

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Zhang, Yu, Hui Li, Yi-Li Min, et al. "Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system." Science Advances 6, no. 8 (2020): eaay6812. http://dx.doi.org/10.1126/sciadv.aay6812.

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Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by mutations in the dystrophin gene (DMD). Previously, we applied CRISPR-Cas9–mediated “single-cut” genome editing to correct diverse genetic mutations in animal models of DMD. However, high doses of adeno-associated virus (AAV) are required for efficient in vivo genome editing, posing challenges for clinical application. In this study, we packaged Cas9 nuclease in single-stranded AAV (ssAAV) and CRISPR single guide RNAs in self-complementary AAV (scAAV) and delivered this dual AAV system into a mouse model of DMD. The

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DÖLARSLAN, Melda. "CRISPR-Cas9 Mediated Gene Correction of CFTR Mutations in Cystic Fibrosis: Evaluating Efficacy, Safety, and Long-Term Outcomes in Patient-Derived Lung Organoids." SHIFAA 2023 (May 19, 2023): 1–8. http://dx.doi.org/10.70470/shifaa/2023/005.

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Cystic fibrosis (CF) is a genetic disorder caused by mutations in the *CFTR* gene, leading to severe respiratory and digestive problems. Current therapies including AAV gene therapy, small molecule modifiers, and RNA-based therapies only partially restore *CFTR* function, leaving a need for more effective therapies The aim of this review is to address CRISPR-1. Cas9-mediated genetic correction in patient derived lung organoids Efficacy, safety and long-term outcomes are evaluated as a novel strategy for CF therapy. Our goal was to achieve high gene editing efficiency, restore *CFTR* function t

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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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Walsh, Colin, and Sha Jin. "Induced Pluripotent Stem Cells and CRISPR-Cas9 Innovations for Treating Alpha-1 Antitrypsin Deficiency and Glycogen Storage Diseases." Cells 13, no. 12 (2024): 1052. http://dx.doi.org/10.3390/cells13121052.

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Human induced pluripotent stem cell (iPSC) and CRISPR-Cas9 gene-editing technologies have become powerful tools in disease modeling and treatment. By harnessing recent biotechnological advancements, this review aims to equip researchers and clinicians with a comprehensive and updated understanding of the evolving treatment landscape for metabolic and genetic disorders, highlighting how iPSCs provide a unique platform for detailed pathological modeling and pharmacological testing, driving forward precision medicine and drug discovery. Concurrently, CRISPR-Cas9 offers unprecedented precision in

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Amoasii, Leonela, Chengzu Long, Hui Li, et al. "Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy." Science Translational Medicine 9, no. 418 (2017): eaan8081. http://dx.doi.org/10.1126/scitranslmed.aan8081.

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Duchenne muscular dystrophy (DMD) is a severe, progressive muscle disease caused by mutations in the dystrophin gene. The majority of DMD mutations are deletions that prematurely terminate the dystrophin protein. Deletions of exon 50 of the dystrophin gene are among the most common single exon deletions causing DMD. Such mutations can be corrected by skipping exon 51, thereby restoring the dystrophin reading frame. Using clustered regularly interspaced short palindromic repeats/CRISPR-associated 9 (CRISPR/Cas9), we generated a DMD mouse model by deleting exon 50. These ΔEx50 mice displayed sev

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Charpentier, Emmanuelle. "Gene Editing and Genome Engineering with CRISPR-Cas9." Molecular Frontiers Journal 01, no. 02 (2017): 99–107. http://dx.doi.org/10.1142/s2529732517400119.

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The RNA-programmable CRISPR-Cas9 technology allows precise and efficient engineering or correction of mutations, modulation of gene expression and marking of DNA in a wide variety of cell types and organisms in the three domains of life. Because of its versatility and ease of design, this powerful technology has been rapidly and universally adopted for genome editing applications in life science research. It is also recognized for its promising and potentially transformative applications in biotechnology, medicine and agriculture.

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Padayachee, Jananee, and Moganavelli Singh. "Therapeutic applications of CRISPR/Cas9 in breast cancer and delivery potential of gold nanomaterials." Nanobiomedicine 7 (January 1, 2020): 184954352098319. http://dx.doi.org/10.1177/1849543520983196.

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Globally, approximately 1 in 4 cancers in women are diagnosed as breast cancer (BC). Despite significant advances in the diagnosis and therapy BCs, many patients develop metastases or relapses. Hence, novel therapeutic strategies are required, that can selectively and efficiently kill malignant cells. Direct targeting of the genetic and epigenetic aberrations that occur in BC development is a promising strategy to overcome the limitations of current therapies, which target the tumour phenotype. The clustered regularly interspaced short palindromic repeats (CRISPR)/Cas system, composed of only

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Wade, Mark. "High-Throughput Silencing Using the CRISPR-Cas9 System." Journal of Biomolecular Screening 20, no. 8 (2015): 1027–39. http://dx.doi.org/10.1177/1087057115587916.

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The clustered regularly interspaced short palindromic repeats (CRISPR)/Cas system has been seized upon with a fervor enjoyed previously by small interfering RNA (siRNA) and short hairpin RNA (shRNA) technologies and has enormous potential for high-throughput functional genomics studies. The decision to use this approach must be balanced with respect to adoption of existing platforms versus awaiting the development of more “mature” next-generation systems. Here, experience from siRNA and shRNA screening plays an important role, as issues such as targeting efficiency, pooling strategies, and off

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Bravo, Jack P. K., Mu-Sen Liu, Grace N. Hibshman, et al. "Publisher Correction: Structural basis for mismatch surveillance by CRISPR–Cas9." Nature 604, no. 7904 (2022): E10. http://dx.doi.org/10.1038/s41586-022-04655-8.

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Schaefer, Kellie A., Wen-Hsuan Wu, Diana F. Colgan, Stephen H. Tsang, Alexander G. Bassuk, and Vinit B. Mahajan. "Correction: Retraction: Unexpected mutations after CRISPR–Cas9 editing in vivo." Nature Methods 15, no. 5 (2018): 394. http://dx.doi.org/10.1038/nmeth0518-394a.

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Kawashima, Nozomu, Yusuke Okuno, Yuko Sekiya, et al. "Correction of Fanconi Anemia Mutation Using the Crispr/Cas9 System." Blood 126, no. 23 (2015): 3622. http://dx.doi.org/10.1182/blood.v126.23.3622.3622.

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Abstract Introduction Gene therapy has been developed for genetic diseases, either to restore normal function for loss-of-function mutations or to inhibit gain-of-function mutations. Gene addition using genetically engineered viral and plasmid vectors has successfully corrected cell pathophysiology resulting in the production of functional proteins. Therapeutic safety has been reinforced by the use of self-inactivating vectors; however, the potential risk of tumorigenesis raises concerns for insertional mutagenesis combined with acquired somatic mutations. Recent advances in gene editing using

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Li, Dandan, Minglin Ou, Wei Zhang, et al. "CRISPR/Cas9-Mediated Gene Correction in Osteopetrosis Patient-Derived iPSCs." Frontiers in Bioscience-Landmark 28, no. 6 (2023): 131. http://dx.doi.org/10.31083/j.fbl2806131.

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MEHAR, A., M. GHAFFAR, S. MUSTAFA, S. IQBAL, B. MASOOD, and I. SHAHID. "THERAPEUTIC CRISPR/CAS9 GENOME EDITING TOOL FOR TREATING SICKLE CELL DISEASE." Biological and Clinical Sciences Research Journal 2024, no. 1 (2024): 987. http://dx.doi.org/10.54112/bcsrj.v2024i1.987.

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Sickle cell disease (SCD) is a serious inherited condition that leads to high rates of illness and mortality, and a complete cure is not yet available. Recent advances suggest that correcting the genetic mutations in hematopoietic stem/progenitor cells (HSPCs) or boosting fetal hemoglobin might stop red blood cells from sickling. Techniques like CRISPR/Cas9 genome editing and creating induced pluripotent stem cells (iPSCs) are being explored to address SCD more effectively. Genome editing has proved beneficial as a treatment choice. CRISPR/Cas9's ongoing revelations have disrupted genetic desi

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Jung, Hyerin, Yeri Alice Rim, Narae Park, Yoojun Nam, and Ji Hyeon Ju. "Restoration of Osteogenesis by CRISPR/Cas9 Genome Editing of the Mutated COL1A1 Gene in Osteogenesis Imperfecta." Journal of Clinical Medicine 10, no. 14 (2021): 3141. http://dx.doi.org/10.3390/jcm10143141.

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Osteogenesis imperfecta (OI) is a genetic disease characterized by bone fragility and repeated fractures. The bone fragility associated with OI is caused by a defect in collagen formation due to mutation of COL1A1 or COL1A2. Current strategies for treating OI are not curative. In this study, we generated induced pluripotent stem cells (iPSCs) from OI patient-derived blood cells harboring a mutation in the COL1A1 gene. Osteoblast (OB) differentiated from OI-iPSCs showed abnormally decreased levels of type I collagen and osteogenic differentiation ability. Gene correction of the COL1A1 gene usin

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Wada, Umar Bello, Shanti Nath, Mohammad Zeeshan, Kushiram Sharma, and Yogesh Joshi. "Progress on CRISPR -Cas9 Gene Editing Technology in Sickle cell disease: A Review." Galore International Journal of Health Sciences and Research 9, no. 4 (2025): 55–62. https://doi.org/10.52403/gijhsr.20240404.

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Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas9 system as a method of Gene editing possess a broad range of applications for genetic modification, diagnosis and treatment for curing non-infectious (such as the Sickle cell disease) as well as infectious diseases. Initially identified in bacteria and archaea to fight plasmids' DNA and/or bacteriophage infecting bacteria. Cas9 and gRNA forms a complex to target and cleave the desired gene, providing defense (adaptive immunity) against viral infections to the host, also allow bacteria to recognize genetic sequences using sp

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Han, Xin, Zongbin Liu, Myeong chan Jo, et al. "CRISPR-Cas9 delivery to hard-to-transfect cells via membrane deformation." Science Advances 1, no. 7 (2015): e1500454. http://dx.doi.org/10.1126/sciadv.1500454.

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The CRISPR (clustered regularly interspaced short palindromic repeats)–Cas (CRISPR-associated) nuclease system represents an efficient tool for genome editing and gene function analysis. It consists of two components: single-guide RNA (sgRNA) and the enzyme Cas9. Typical sgRNA and Cas9 intracellular delivery techniques are limited by their reliance on cell type and exogenous materials as well as their toxic effects on cells (for example, electroporation). We introduce and optimize a microfluidic membrane deformation method to deliver sgRNA and Cas9 into different cell types and achieve success

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Beretta, Maxime, and Hugo Mouquet. "Ingénierie de lymphocytes B humains produisant des anticorps neutralisant le virus VIH-1 par édition génique CRISPR-Cas9." médecine/sciences 35, no. 12 (2019): 993–96. http://dx.doi.org/10.1051/medsci/2019196.

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Les anticorps (ou immunoglobulines, Ig) produits par les lymphocytes B sont essentiels aux réponses immunitaires induites par les infections et les vaccins. Les anticorps sont des glycoprotéines hétérodimériques résultant de l’association de deux chaînes lourdes (IgH), et de deux chaînes légères (IgL) d’immunoglobuline. Les chaînes IgH et IgL possèdent des régions « hypervariables », également appelées en anglais complementarity determining regions (CDR), situées dans leurs domaines variables, VH et VL, qui, en se combinant, forment le site de liaison à l’antigène ou paratope.

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Vibhuti Choubisa and Sunil Sharma. "Unveiling neural network potential in forecasting CRISPR effects and off-target prophecies for gene editing." International Journal of Science and Research Archive 10, no. 1 (2023): 252–59. http://dx.doi.org/10.30574/ijsra.2023.10.1.0738.

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The revolutionary Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) - associated protein 9 (Cas9) systems has emerged as a groundbreaking gene-editing tool, widely embraced within biomedical research. Nonetheless, the utilization of guide RiboNucleic Acids (gRNAs) in the CRISPR-Cas9 system can inadvertently trigger undesired off-target effects, consequently impinging on the practical implementation of this technique. Existing in silico prediction methods that focus on off-target effects have exhibited constrained predictive accuracy, necessitating further enhancement. To tackl

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Miki, Toshio, Ludivina Vazquez, Lisa Yanuaria, et al. "Induced Pluripotent Stem Cell Derivation and Ex Vivo Gene Correction Using a Mucopolysaccharidosis Type 1 Disease Mouse Model." Stem Cells International 2019 (April 1, 2019): 1–10. http://dx.doi.org/10.1155/2019/6978303.

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Mucopolysaccharidosis type 1 (MPS-1), also known as Hurler’s disease, is a congenital metabolic disorder caused by a mutation in the alpha-L-iduronidase (IDUA) gene, which results in the loss of lysosomal enzyme function for the degradation of glycosaminoglycans. Here, we demonstrate the proof of concept of ex vivo gene editing therapy using induced pluripotent stem cell (iPSC) and CRISPR/Cas9 technologies with MPS-1 model mouse cell. Disease-affected iPSCs were generated from Idua knockout mouse embryonic fibroblasts, which carry a disrupting neomycin-resistant gene cassette (Neor) in exon VI

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Alanis-Lobato, Gregorio, Jasmin Zohren, Afshan McCarthy, et al. "Frequent loss of heterozygosity in CRISPR-Cas9–edited early human embryos." Proceedings of the National Academy of Sciences 118, no. 22 (2021): e2004832117. http://dx.doi.org/10.1073/pnas.2004832117.

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CRISPR-Cas9 genome editing is a promising technique for clinical applications, such as the correction of disease-associated alleles in somatic cells. The use of this approach has also been discussed in the context of heritable editing of the human germ line. However, studies assessing gene correction in early human embryos report low efficiency of mutation repair, high rates of mosaicism, and the possibility of unintended editing outcomes that may have pathologic consequences. We developed computational pipelines to assess single-cell genomics and transcriptomics datasets from OCT4 (POU5F1) CR

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Cosenza, Lucia Carmela, Cristina Zuccato, Matteo Zurlo, Roberto Gambari та Alessia Finotti. "Co-Treatment of Erythroid Cells from β-Thalassemia Patients with CRISPR-Cas9-Based β039-Globin Gene Editing and Induction of Fetal Hemoglobin". Genes 13, № 10 (2022): 1727. http://dx.doi.org/10.3390/genes13101727.

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Gene editing (GE) is an efficient strategy for correcting genetic mutations in monogenic hereditary diseases, including β-thalassemia. We have elsewhere reported that CRISPR-Cas9-based gene editing can be employed for the efficient correction of the β039-thalassemia mutation. On the other hand, robust evidence demonstrates that the increased production of fetal hemoglobin (HbF) can be beneficial for patients with β-thalassemia. The aim of our study was to verify whether the de novo production of adult hemoglobin (HbA) using CRISPR-Cas9 gene editing can be combined with HbF induction protocols.

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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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Ababneh, Nidaa A., Jakub Scaber, Rowan Flynn, et al. "Correction of amyotrophic lateral sclerosis related phenotypes in induced pluripotent stem cell-derived motor neurons carrying a hexanucleotide expansion mutation in C9orf72 by CRISPR/Cas9 genome editing using homology-directed repair." Human Molecular Genetics 29, no. 13 (2020): 2200–2217. http://dx.doi.org/10.1093/hmg/ddaa106.

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Abstract The G4C2 hexanucleotide repeat expansion (HRE) in C9orf72 is the commonest cause of familial amyotrophic lateral sclerosis (ALS). A number of different methods have been used to generate isogenic control lines using clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 and non-homologous end-joining by deleting the repeat region, with the risk of creating indels and genomic instability. In this study, we demonstrate complete correction of an induced pluripotent stem cell (iPSC) line derived from a C9orf72-HRE positive ALS/frontotemporal dementia patient using CRISPR/

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Chen, Chiao-Lin, Jonathan Rodiger, Verena Chung, et al. "SNP-CRISPR: A Web Tool for SNP-Specific Genome Editing." G3: Genes|Genomes|Genetics 10, no. 2 (2019): 489–94. http://dx.doi.org/10.1534/g3.119.400904.

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CRISPR-Cas9 is a powerful genome editing technology in which a single guide RNA (sgRNA) confers target site specificity to achieve Cas9-mediated genome editing. Numerous sgRNA design tools have been developed based on reference genomes for humans and model organisms. However, existing resources are not optimal as genetic mutations or single nucleotide polymorphisms (SNPs) within the targeting region affect the efficiency of CRISPR-based approaches by interfering with guide-target complementarity. To facilitate identification of sgRNAs (1) in non-reference genomes, (2) across varying genetic ba

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Sharma, Saksham, Dhruv Gandhi, Bhoomi Bagadia, Kevin Lee, and Anna Jobilal. "Unravelling fragile minds: the promise of CRISPR and RNA therapy in Fragile X Syndrome." InterConf, no. 48(213) (August 19, 2024): 226–37. http://dx.doi.org/10.51582/interconf.19-20.08.2024.020.

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Background: Fragile X Syndrome (FXS) is caused by the deficiency or absence of the fragile X mental retardation protein (FMRP) due to CGG trinucleotide repeat expansions in the FMR1 gene. This results in cognitive impairments, and behavioural abnormalities, and is a leading monogenic cause of autism spectrum disorder. Despite extensive research, effective treatments remain limited, prompting exploration into advanced genetic therapies such as CRISPR/Cas9 gene editing and RNA-based therapies. Methods: A systematic search was conducted using the PICO framework to identify studies involving indiv

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Salah Uddin, Iffat Munir, Syeda Hina Shah, Afshan Khalid, Sana Barkat Ali, and Mohammad Rayyan Faisal. "A Comparative Analysis of CRISPR-Cas9, Base Editing and Prime Editing Technologies for Precision Gene Therapy in Cardiac Disease Applications." Indus Journal of Bioscience Research 3, no. 4 (2025): 687–96. https://doi.org/10.70749/ijbr.v3i4.1173.

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This study provides a comparative analysis of CRISPR-Cas9, base editing, and prime editing technologies based on their potential applications in precision gene therapy for genetic heart diseases. The primary objective was to compare the efficiency, accuracy, and safety of these technologies in editing cardiomyocyte genes and their implications for the treatment of cardiac diseases. Employing induced pluripotent stem cell (iPSC)-derived cardiomyocytes, we compared editing efficiency, off-target mutations, cell viability, electrophysiological properties, and protein expression following gene edi

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Pavani, Giulia, Anna Fabiano, Marine Laurent та ін. "Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells". Blood Advances 5, № 5 (2021): 1137–53. http://dx.doi.org/10.1182/bloodadvances.2020001996.

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Abstract β-thalassemias (β-thal) are a group of blood disorders caused by mutations in the β-globin gene (HBB) cluster. β-globin associates with α-globin to form adult hemoglobin (HbA, α2β2), the main oxygen-carrier in erythrocytes. When β-globin chains are absent or limiting, free α-globins precipitate and damage cell membranes, causing hemolysis and ineffective erythropoiesis. Clinical data show that severity of β-thal correlates with the number of inherited α-globin genes (HBA1 and HBA2), with α-globin gene deletions having a beneficial effect for patients. Here, we describe a novel strateg

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Joung, Julia, Silvana Konermann, Jonathan S. Gootenberg, et al. "Author Correction: Genome-scale CRISPR-Cas9 knockout and transcriptional activation screening." Nature Protocols 14, no. 7 (2018): 2259. http://dx.doi.org/10.1038/s41596-018-0063-0.

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Morishige, Satoshi, Shinichi Mizuno, Hidetoshi Ozawa, et al. "CRISPR/Cas9-mediated gene correction in hemophilia B patient-derived iPSCs." International Journal of Hematology 111, no. 2 (2019): 225–33. http://dx.doi.org/10.1007/s12185-019-02765-0.

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Min, Yi-Li, Hui Li, Cristina Rodriguez-Caycedo, et al. "CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells." Science Advances 5, no. 3 (2019): eaav4324. http://dx.doi.org/10.1126/sciadv.aav4324.

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Mutations in the dystrophin gene cause Duchenne muscular dystrophy (DMD), which is characterized by lethal degeneration of cardiac and skeletal muscles. Mutations that delete exon 44 of the dystrophin gene represent one of the most common causes of DMD and can be corrected in ~12% of patients by editing surrounding exons, which restores the dystrophin open reading frame. Here, we present a simple and efficient strategy for correction of exon 44 deletion mutations by CRISPR-Cas9 gene editing in cardiomyocytes obtained from patient-derived induced pluripotent stem cells and in a new mouse model

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Öktem, Mert, Enrico Mastrobattista, and Olivier G. de Jong. "Amphipathic Cell-Penetrating Peptide-Aided Delivery of Cas9 RNP for In Vitro Gene Editing and Correction." Pharmaceutics 15, no. 10 (2023): 2500. http://dx.doi.org/10.3390/pharmaceutics15102500.

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The therapeutic potential of the CRISPR-Cas9 gene editing system in treating numerous genetic disorders is immense. To fully realize this potential, it is crucial to achieve safe and efficient delivery of CRISPR-Cas9 components into the nuclei of target cells. In this study, we investigated the applicability of the amphipathic cell-penetrating peptide LAH5, previously employed for DNA delivery, in the intracellular delivery of spCas9:sgRNA ribonucleoprotein (RNP) and the RNP/single-stranded homology-directed repair (HDR) template. Our findings reveal that the LAH5 peptide effectively formed na

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Pavlova, Sophia V., Angelina E. Shulgina, Julia M. Minina, Suren M. Zakian, and Elena V. Dementyeva. "Generation of Isogenic iPSC Lines for Studying the Effect of the p.N515del (c.1543_1545delAAC) Variant on MYBPC3 Function and Hypertrophic Cardiomyopathy Pathogenesis." International Journal of Molecular Sciences 25, no. 23 (2024): 12900. https://doi.org/10.3390/ijms252312900.

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The clinical significance of numerous cardiovascular gene variants remains to be determined. CRISPR/Cas9 allows for the introduction and/or correction of a certain variant in induced pluripotent stem cells (iPSCs). The resulting isogenic iPSC lines can be differentiated into cardiomyocytes and used as a platform to assess the pathogenicity of the variant. In this study, isogenic iPSC lines were generated for a variant of unknown significance found previously in a patient with hypertrophic cardiomyopathy (HCM), p.N515del (c.1543_1545delAAC) in MYBPC3. The deletion was corrected with CRISPR/Cas9

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Luo, Xianjin, Eric Weidinger, Tobias Burghardt, Miriam Höhn, and Ernst Wagner. "CRISPR/Cas9 Ribonucleoprotein Delivery Enhanced by Lipo-Xenopeptide Carriers and Homology-Directed Repair Modulators: Insights from Reporter Cell Lines." International Journal of Molecular Sciences 26, no. 9 (2025): 4361. https://doi.org/10.3390/ijms26094361.

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CRISPR-Cas9 genome editing is a versatile platform for studying and treating various diseases. Homology-directed repair (HDR) with DNA donor templates serves as the primary pathway for gene correction in therapeutic applications, but its efficiency remains a significant challenge. This study investigates strategies to enhance gene correction efficiency using a T-shaped lipo-xenopeptide (XP)-based Cas9 RNP/ssDNA delivery system combined with various HDR enhancers. Nu7441, a known DNA-PKcs inhibitor, was found to be most effective in enhancing HDR-mediated gene correction. An over 10-fold increa

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Gobalakrishnan, Krishshan, Vignesh Jayarajan, Veronica Kinsler, and Wei-Li Di. "O18 Precision genome editing for targeted correction of pathogenic D50N mutation in keratitis–ichthyosis–deafness syndrome using CRISPR/Cas9 and homology-directed repair." British Journal of Dermatology 190, no. 6 (2024): e76-e77. http://dx.doi.org/10.1093/bjd/ljae105.018.

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Abstract Introduction and aims Keratitis–ichthyosis–deafness (KID) syndrome is an ectodermal disorder that causes blindness, skin inflammation and deafness. It is caused by autosomal dominant mutations in the gap junction beta 2 (GJB2) gene with 86% being a hotspot mutation on c.148G&gt;A, resulting in the amino acid replacement (D50N) in its coding protein connexin 26 (Cx26). There is currently no curative treatment for KID syndrome. We aim to correct the hotspot mutation D50N using a genome-editing approach. Methods Keratinocytes generated from the patient with KID who had a D50N mutatio

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