title: Current Progress in Therapeutic Gene Editing for Monogenic Diseases.
abstract: 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.
authors.affiliation: School of Biological Sciences, Royal Holloway, University of London, Egham, Surrey, UK.
authors.forename: Versha
authors.initials: V
authors.lastname: Prakash
authors.affiliation: School of Biological Sciences, Royal Holloway, University of London, Egham, Surrey, UK.
authors.forename: Marc
authors.initials: M
authors.lastname: Moore
authors.affiliation: School of Biological Sciences, Royal Holloway, University of London, Egham, Surrey, UK.
authors.forename: Rafael J
authors.initials: RJ
authors.lastname: Yáñez-Muñoz
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D004353:Drug Evaluation, Preclinical; D000072669:Gene Editing / Q000379:methods; D018390:Gene Targeting; D018014:Gene Transfer Techniques; D030342:Genetic Diseases, Inborn / Q000235:genetics* / Q000628:therapy*; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D057170:Translational Research, Biomedical