Gene-targeting pharmaceuticals for single-gene disorders

Human Molecular Genetics

Volumn 25, Issue R1, 2016, Pages R18-R26

  Beaudet, Arthur L ^a   Meng, Linyan ^a  

^a BAYLOR COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; SMALL INTERFERING RNA; TRANSTHYRETIN;

AMYLOIDOSIS; ATTR AMYLOIDOSIS; CRISPR-CAS9 SYSTEM; CYSTIC FIBROSIS; DOWN SYNDROME; DUCHENNE MUSCULAR DYSTROPHY; EPIGENETICS; EXON SKIPPING; GENE EXPRESSION; GENE TARGETING; HAPPY PUPPET SYNDROME; HUMAN; HUNTINGTON CHOREA; HYPERCHOLESTEROLEMIA; MONOGENIC DISORDER; NONSENSE MUTATION; OLIGONUCLEOTIDE THERAPY; PHARMACEUTICS; REVIEW; RNA SPLICING; SPINAL MUSCULAR ATROPHY; SPINOCEREBELLAR DEGENERATION; GENETIC DISEASES, INBORN; RARE DISEASES;

GENE TARGETING; GENETIC DISEASES, INBORN; HUMANS; RARE DISEASES;

EID: 84978362015     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddv476     Document Type: Review

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