siRNA knock-down of mutant torsinA restores processing through secretory pathway in DYT1 dystonia cells

Human Molecular Genetics

Volumn 17, Issue 10, 2008, Pages 1436-1445

  Hewett, Jeffrey W ^a,b   Nery, Flávia C ^a,b   Niland, Brian ^a,b   Ge, Pei ^c   Tan, Pamela ^c   Hadwiger, Philipp ^c   Tannous, Bakhos A ^a,b   Sah, Dinah W Y ^c   Breakefield, Xandra O ^a,b  

^a HARVARD MEDICAL SCHOOL   (United States)

^b MASSACHUSETTS GENERAL HOSPITAL   (United States)

^c ALNYLAM PHARMACEUTICALS   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE TRIPHOSPHATASE; GLUTAMIC ACID; GLYCERALDEHYDE 3 PHOSPHATE DEHYDROGENASE; LENTIVIRUS VECTOR; LUCIFERASE; MESSENGER RNA; SMALL INTERFERING RNA; TORSINA;

ALLELE; ARTICLE; CARBOXY TERMINAL SEQUENCE; CELL NUCLEUS MEMBRANE; CONTROLLED STUDY; DOMINANT INHERITANCE; DOWN REGULATION; ENDOPLASMIC RETICULUM; FIBROBLAST; GENE MUTATION; GENETIC TRANSDUCTION; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; MEMBRANE STRUCTURE; MOTOR DYSFUNCTION; PATHOGENESIS; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; PROTEIN PROCESSING; PROTEIN SECRETION; RNA INTERFERENCE; TORSION DYSTONIA;

BASE SEQUENCE; CELL CULTURE TECHNIQUES; CELLS, CULTURED; DYSTONIA MUSCULORUM DEFORMANS; FIBROBLASTS; GENE SILENCING; GENES, REPORTER; HUMANS; LUCIFERASES; MOLECULAR CHAPERONES; MOLECULAR SEQUENCE DATA; MUTATION; PROTEIN PROCESSING, POST-TRANSLATIONAL; PROTEIN TRANSPORT; RNA, MESSENGER; RNA, SMALL INTERFERING; TRANSFECTION;

LENTIVIRUS;

EID: 43049088486     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddn032     Document Type: Article

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