Gene editing for duchenne muscular dystrophy using the CRISPR/Cas9 technology: The importance of fine-tuning the approach

Molecular Therapy

Volumn 24, Issue 11, 2016, Pages 1888-1889

  Tremblay, Jacques P ^a   Iyombe Engembe, Jean Paul ^a   Duchêne, Benjamin ^a   Ouellet, Dominique L ^a  

^a Université Laval   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; MESSENGER RNA;

BECKER MUSCULAR DYSTROPHY; CRISPR CAS SYSTEM; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; GENE DELETION; GENE EDITING; GENE EXPRESSION; GENE MUTATION; GENE STRUCTURE; GENETIC ENGINEERING; HUMAN; INTRON; LETTER; PROTEIN EXPRESSION; ANIMAL; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; GENETICS; MOUSE; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE; AMINO TERMINAL SEQUENCE; CARBOXY TERMINAL SEQUENCE; CRISPR-CAS9 SYSTEM; DISEASE SEVERITY; DMD GENE; DNA SEQUENCE; DOUBLE STRANDED DNA BREAK; EXON; IMMUNE RESPONSE; NONHUMAN; NONSENSE MUTATION; PHENOTYPE;

ANIMALS; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS; CRISPR-CAS SYSTEMS; DYSTROPHIN; GENE EDITING; HUMANS; MICE; MICE, INBRED MDX; MUSCULAR DYSTROPHY, DUCHENNE;

EID: 85002844185     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2016.191     Document Type: Letter

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