Current progress in therapeutic gene editing for monogenic diseases

Molecular Therapy

Volumn 24, Issue 3, 2016, Pages 465-474

  Prakash, Versha ^a   Moore, Marc ^a   Yáñez Muñoz, Rafael J ^a  

^a UNIVERSITY OF LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; ZINC FINGER NUCLEASE;

BETA THALASSEMIA; BONE MARROW DISEASE; CRISPR CAS SYSTEM; CYSTIC FIBROSIS; DNA END JOINING REPAIR; DNA REPAIR; DUCHENNE MUSCULAR DYSTROPHY; GENE EDITING; GENE MUTATION; GENE THERAPY; HEMOPHILIA; HOMOLOGY DIRECTED REPAIR; HUMAN; IMMUNE DEFICIENCY; MONOGENIC DISORDER; NONHUMAN; REVIEW; SICKLE CELL ANEMIA; ANIMAL; DISEASE MODEL; GENE TARGETING; GENE TRANSFER; GENETIC DISEASES, INBORN; GENETICS; PRECLINICAL STUDY; PROCEDURES; TRANSLATIONAL RESEARCH;

ANIMALS; DISEASE MODELS, ANIMAL; DRUG EVALUATION, PRECLINICAL; GENE EDITING; GENE TARGETING; GENE TRANSFER TECHNIQUES; GENETIC DISEASES, INBORN; GENETIC THERAPY; HUMANS; TRANSLATIONAL MEDICAL RESEARCH;

EID: 84960327653     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2016.5     Document Type: Review

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