Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Nature Medicine

Volumn 12, Issue 3, 2006, Pages 342-347

  Manno, Catherine S ^a,b   Arruda, Valder R ^a,b   Pierce, Glenn F ^c   Glader, Bertil ^d   Ragni, Margaret ^e   Rasko, John ^f   Ozelo, Margareth C ^g   Hoots, Keith ^h   Blatt, Philip ⁱ   Konkle, Barbara ^j   Dake, Michael ^d   Kaye, Robin ^a,b   Razavi, Mahmood ^d   Zajko, Albert ^j   Zehnder, James ^d   Nakai, Hiroyuki ^d   Chew, Amy ^a,c   Leonard, Debra ^b,k   Wright, J Fraser ^c   Lessard, Ruth R ^c   Sommer, Jürg M ^c   Tigges, Michael ^c   Sabatino, Denise ^a   Luk, Alvin ^c   Jiang, Haiyan ^c   Mingozzi, Federico ^a   Couto, Linda ^c   Ertl, Hildegund C ^l   High, Katherine A ^a,c,m   Kay, Mark A ^d   more..

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c Avigen Inc   (United States)

^d STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^e UNIVERSITY OF PITTSBURGH MEDICAL CENTER   (United States)

^f ROYAL PRINCE ALFRED HOSPITAL   (Australia)

^g UNIVERSITY OF CAMPINAS   (Brazil)

^h UNIVERSITY OF TEXAS   (United States)

ⁱ CHRISTIANA HOSPITAL   (United States)

^j PENN PRESBYTERIAN MEDICAL CENTER   (United States)

^k WEILL CORNELL MEDICAL COLLEGE   (United States)

^l WISTAR INSTITUTE   (United States)

^m HOWARD HUGHES MEDICAL INSTITUTE   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; BLOOD CLOTTING FACTOR 9; VIRUS ANTIGEN;

ABNORMALLY HIGH SUBSTRATE CONCENTRATION IN BLOOD; ACUTE TOXICITY; ADULT; ARTICLE; CELL DESTRUCTION; CELLULAR IMMUNITY; CHILL; CLINICAL ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DISEASE SEVERITY; DRUG MEGADOSE; DRUG SAFETY; EMBRYO; FEVER; GENE TARGETING; GENETIC TRANSDUCTION; HEART INFARCTION; HEMATOMA; HEMOPHILIA B; HEPATITIS; HOST; HUMAN; HUMAN CELL; IMMUNOMODULATION; INFUSION; INJECTION SITE REACTION; LIVER CELL; LIVER INJURY; LIVER TOXICITY; MALE; NAUSEA; PARVOVIRUS VECTOR; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; PROTEIN EXPRESSION; SYMPTOM; TRANSAMINITIS; VIRAL GENE THERAPY; VIRUS CAPSID; VOMITING;

ADULT; AMINO ACID SEQUENCE; ANIMALS; DEPENDOVIRUS; DOGS; DOSE-RESPONSE RELATIONSHIP, DRUG; EXONS; FACTOR IX; GENE THERAPY; GENETIC VECTORS; HEMOPHILIA A; HUMANS; INTERFERON TYPE II; INTRONS; LIVER; MALE; MICE; MIDDLE AGED; MOLECULAR SEQUENCE DATA; MONOCYTES; TRANSDUCTION, GENETIC;

CANIS FAMILIARIS; MIRIDAE; PARVOVIRUS;

EID: 33644820684     PISSN: 10788956     EISSN: 1546170X     Source Type: Journal    
DOI: 10.1038/nm1358     Document Type: Article

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