Hereditary inclusion body myopathy: Single patient response to intravenous dosing of GNE gene lipoplex

Human Gene Therapy

Volumn 22, Issue 11, 2011, Pages 1331-1341

  Nemunaitis, Gregory ^a,b   Jay, Chris M ^c   Maples, Phillip B ^c   Gahl, William A ^d   Huizing, Marjan ^d   Yardeni, Tal ^d   Tong, Alex W ^c   Phadke, Anagha P ^c   Pappen, Beena O ^c   Bedell, Cynthia ^a   Allen, Henry ^e   Hernandez, Cathy ^e   Templeton, Nancy S ^c   Kuhn, Joseph ^f   Senzer, Neil ^a,c,g   Nemunaitis, John ^a,c,g  

^a Mary Crowley Cancer Research Centers   (United States)

^b METROHEALTH MEDICAL CENTER   (United States)

^c GRADALIS INC   (United States)

^d NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

^e Hospitalist Internists Associates   (United States)

^f WLS Surgical Associates PA   (United States)

^g TEXAS ONCOLOGY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AMINOTRANSFERASE; LIPOPLEX; MANNOSAMINE; N ACETYLMANNOSAMINE KINASE; PLASMID DNA; SIALIC ACID; UNCLASSIFIED DRUG;

ADULT; ARTICLE; CASE REPORT; DRUG DOSE ESCALATION; DRUG FEVER; FEMALE; FEVER; FORCED VITAL CAPACITY; GENE EXPRESSION; GENE THERAPY; HEREDITARY INCLUSION BODY MYOPATHY; HEREDITY; HUMAN; HUMAN TISSUE; HYPONATREMIA; HYPOTENSION; IMMUNOHISTOCHEMISTRY; MUSCLE FUNCTION; MUSCLE STRENGTH; MYALGIA; MYOPATHY; PLASMID; POLYMERASE CHAIN REACTION; QUADRICEPS FEMORIS MUSCLE; QUANTITATIVE ANALYSIS; SIDE EFFECT; TACHYCARDIA;

ADULT; FEMALE; GENE THERAPY; GENETIC VECTORS; HUMANS; INFUSIONS, INTRAVENOUS; LIPOSOMES; MULTIENZYME COMPLEXES; MUSCLE, SKELETAL; MYOSITIS, INCLUSION BODY; RNA;

EID: 81855184520     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2010.192     Document Type: Article

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