Current Progress in Therapeutic Gene Editing for Monogenic Diseases

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, nucleas...

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Bibliographic Details
Published inMolecular therapy Vol. 24; no. 3; pp. 465 - 474
Main Authors Prakash, Versha, Moore, Marc, Yáñez-Muñoz, Rafael J
Format Journal Article
LanguageEnglish
Published United States Elsevier Inc 01.03.2016
Elsevier Limited
Nature Publishing Group
Subjects
Animals
Disease Models, Animal
Drug Evaluation, Preclinical
Gene Editing - methods
Gene Targeting
Gene Transfer Techniques
Genetic Diseases, Inborn - genetics
Genetic Diseases, Inborn - therapy
Genetic Therapy - methods
Humans
Review
Translational Medical Research
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Summary: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.
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ISSN:1525-0016
1525-0024
DOI:10.1038/mt.2016.5