Current Progress in Therapeutic Gene Editing for Monogenic Diseases

التفاصيل البيبلوغرافية
العنوان: Current Progress in Therapeutic Gene Editing for Monogenic Diseases
المؤلفون: Versha Prakash, Rafael J. Yáñez-Muñoz, Marc Moore
المصدر: Molecular therapy : the journal of the American Society of Gene Therapy. 24(3)
سنة النشر: 2015
مصطلحات موضوعية: 0301 basic medicine, Duchenne muscular dystrophy, Genetic enhancement, Drug Evaluation, Preclinical, Computational biology, Review, Biology, Genome, Genome engineering, Translational Research, Biomedical, 03 medical and health sciences, Genome editing, Drug Discovery, Genetics, medicine, Animals, Humans, Molecular Biology, Pharmacology, Gene Editing, Nuclease, Gene Transfer Techniques, Genetic Diseases, Inborn, Gene targeting, Genetic Therapy, medicine.disease, Non-homologous end joining, Disease Models, Animal, 030104 developmental biology, Gene Targeting, biology.protein, Molecular Medicine
الوصف: Programmable nucleases allow defined alterations in the genome with ease-of-use, efficiency, and specificity. Their availability has led to accurate and widespread genome engineering, with multiple applications in basic research, biotechnology, and therapy. With regard to human gene therapy, nuclease-based gene editing has facilitated development of a broad range of therapeutic strategies based on both nonhomologous end joining and homology-dependent repair. This review discusses current progress in nuclease-based therapeutic applications for a subset of inherited monogenic diseases including cystic fibrosis, Duchenne muscular dystrophy, diseases of the bone marrow, and hemophilia and highlights associated challenges and future prospects.
تدمد: 1525-0024
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::24196500c99f3375986ad036629752c9
https://pubmed.ncbi.nlm.nih.gov/26765770
حقوق: OPEN
رقم الأكسشن: edsair.doi.dedup.....24196500c99f3375986ad036629752c9
قاعدة البيانات: OpenAIRE