Immune Responses to Intramuscular Administration of Alipogene Tiparvovec (AAV1-LPLS447X) in a Phase II Clinical Trial of Lipoprotein Lipase Deficiency Gene Therapy-Reference-Cited by-同舟云学术

Immune Responses to Intramuscular Administration of Alipogene Tiparvovec (AAV1-LPLS447X) in a Phase II Clinical Trial of Lipoprotein Lipase Deficiency Gene Therapy

Published:2014-03 Issue:3 Volume:25 Page:180-188
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Ferreira Valerie¹,Twisk Jaap¹,Kwikkers Karin¹,Aronica Eleonora²,Brisson Diane³,Methot Julie³,Petry Harald¹,Gaudet Daniel³

Affiliation:

1. Research and Development, uniQure B.V., 1105 BA Amsterdam, The Netherlands.

2. Department of Neuropathology, Academic Medical Center, 1105 AZ Amsterdam, The Netherlands.

3. Department of Medicine, ECOGENE-21 Clinical Research Center, Universite de Montreal, Chicoutimi Hospital, Chicoutimi, QC G7H 7P2, Quebec, Canada.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2013.169

Reference31 articles.

1. INCREASED EXPRESSION OF HLA ABC CLASS I ANTIGENS BY MUSCLE FIBRES IN DUCHENNE MUSCULAR DYSTROPHY, INFLAMMATORY MYOPATHY, AND OTHER NEUROMUSCULAR DISORDERS

2. Congenital disorder of glycosylation type Ia: a clinicopathological report of a newborn infant with cerebellar pathology

3. Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

4. Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Serotype 2α1-Antitrypsin (AAT) Vector in AAT-Deficient Adults

5. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

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