Ameliorating pathogenesis by removing an exon containing a missense mutation: A potential exon-skipping therapy for laminopathies

Gene Therapy

Volumn 22, Issue 6, 2015, Pages 503-515

  Scharner, J ^a   Figeac, N ^a   Ellis, J A ^a   Zammit, P S ^a  

^a KING'S COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; EMERIN; LAMIN A; LAMIN B; LAMIN; MEMBRANE PROTEIN; NUCLEAR PROTEIN;

ALPHA HELIX; ANIMAL CELL; ARTICLE; CONTROLLED STUDY; EXON; EXON SKIPPING; HUMAN; HUMAN CELL; LAMINOPATHY; MISSENSE MUTATION; MOUSE; NONHUMAN; PATHOGENESIS; PRIORITY JOURNAL; PROTEIN FUNCTION; PROTEIN LOCALIZATION; ANIMAL; C57BL MOUSE; GENE THERAPY; GENE VECTOR; GENETICS; METABOLISM; MUSCULAR DISEASES;

MURINAE;

ANIMALS; EXONS; GENETIC THERAPY; GENETIC VECTORS; LAMINS; MEMBRANE PROTEINS; MICE, INBRED C57BL; MUSCULAR DISEASES; MUTATION, MISSENSE; NUCLEAR PROTEINS;

EID: 84930482160     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2015.8     Document Type: Article

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