The AAV-mediated and RNA-guided CRISPR/Cas9 system for gene therapy of DMD and BMD

Brain and Development

Volumn 39, Issue 7, 2017, Pages 547-556

  Wang, Jing Zhang ^a   Wu, Peng ^a   Shi, Zhi Min ^a   Xu, Yan Li ^a   Liu, Zhi Jun ^a  

^a HEBEI UNIVERSITY OF ENGINEERING   (China)

Author keywords

Adeno associated virus vector; Becker muscular dystrophy; CRISPR Cas9; Duchenne muscular dystrophy; Dystrophin; Gene therapy

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; CAS9 PROTEIN; CRISPR ASSOCIATED PROTEIN; DYSTROPHIN; GUIDED RNA; MESSENGER RNA; RNA; UNCLASSIFIED DRUG;

BECKER MUSCULAR DYSTROPHY; BLOOD BRAIN BARRIER; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; CRISPR-CAS9 SYSTEM; DUCHENNE MUSCULAR DYSTROPHY; EXON; HEART FUNCTION; IMMUNE RESPONSE; NONHUMAN; NONSENSE MUTATION; PERSONALIZED MEDICINE; REVIEW; SKELETAL MUSCLE; STOP CODON; VIRAL GENE THERAPY; ANIMAL; CRISPR CAS SYSTEM; DEPENDOPARVOVIRUS; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN; MALE; PROCEDURES;

ANIMALS; CRISPR-CAS SYSTEMS; DEPENDOVIRUS; DYSTROPHIN; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MALE; MUSCULAR DYSTROPHY, DUCHENNE;

EID: 85017102833     PISSN: 03877604     EISSN: 18727131     Source Type: Journal    
DOI: 10.1016/j.braindev.2017.03.024     Document Type: Review

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