Genome-editing technologies for gene and cell therapy

Molecular Therapy

Volumn 24, Issue 3, 2016, Pages 430-446

  Maeder, Morgan L ^a   Gersbach, Charles A ^b,c,d  

^a EDITAS MEDICINE   (United States)

^b Duke University   (United States)

^c DUKE UNIVERSITY   (United States)

^d DUKE UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTIINFECTIVE AGENT; ANTIVIRUS AGENT; MEGANUCLEASE; NUCLEASE; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; UNCLASSIFIED DRUG; ZINC FINGER NUCLEASE; ENDONUCLEASE;

AMYOTROPHIC LATERAL SCLEROSIS; BETA THALASSEMIA; CANCER IMMUNOTHERAPY; CELL THERAPY; CYSTIC FIBROSIS; DUCHENNE MUSCULAR DYSTROPHY; ENZYME SPECIFICITY; EPIDERMOLYSIS BULLOSA DYSTROPHICA; FRIEDREICH ATAXIA; GENE CORRECTION; GENE DELETION; GENE INSERTION; GENE THERAPY; GENETIC PARAMETERS; GENOME EDITING; HUMAN; HUNTINGTON CHOREA; LEBER CONGENITAL AMAUROSIS; LIMB GIRDLE MUSCULAR DYSTROPHY; MOLECULAR GENETICS; MUTATIONAL ANALYSIS; NONHUMAN; NONVIRAL GENE THERAPY; REVIEW; SICKLE CELL ANEMIA; SPINAL MUSCULAR ATROPHY; STRUCTURE ANALYSIS; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; ANIMAL; BIOLOGICAL THERAPY; GENE EDITING; GENE TARGETING; GENE TRANSFER; GENE VECTOR; GENETICS; GENOME; METABOLISM; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PRECLINICAL STUDY; PROCEDURES;

ANIMALS; CELL- AND TISSUE-BASED THERAPY; CLINICAL TRIALS, PHASE I AS TOPIC; CLINICAL TRIALS, PHASE II AS TOPIC; DRUG EVALUATION, PRECLINICAL; ENDONUCLEASES; GENE EDITING; GENE TARGETING; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; GENOME; HUMANS;

EID: 84960431733     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2016.10     Document Type: Review

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