From Mutation Identification to Therapy: Discovery and Origins of the First Approved Gene Therapy in the Western World-Reference-Cited by-同舟云学术

From Mutation Identification to Therapy: Discovery and Origins of the First Approved Gene Therapy in the Western World

Published:2013-05 Issue:5 Volume:24 Page:472-478
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Kastelein John J.P.¹,Ross Colin J.D.²³⁴,Hayden Michael R.³⁴

Affiliation:

1. Department of Vascular Medicine, Academic Medical Centre, University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.

2. Department of Pediatrics, University of British Columbia, Vancouver, British Columbia V5Z-4H4, Canada.

3. Centre for Molecular Medicine and Therapeutics, Child and Family Research Institute, University of British Columbia, Vancouver, British Columbia V5Z-4H4, Canada.

4. Department of Medical Genetics, University of British Columbia, Vancouver, British Columbia V5Z-4H4, Canada.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2013.063

Reference63 articles.

1. Prevalence, geographical distribution and genealogical investigations of mutation 188 of lipoprotein lipase gene in the French Canadian population of Québec

2. Homozygosity for a mutation in the lipoprotein lipase gene (Gly139?Ser) causes chylomicronaemia in a boy of Spanish descent

3. Compound heterozygosity for a known and a novel defect in the lipoprotein lipase gene (Asp250 ? Asn; Ser251 ? Cys) resulting in lipoprotein lipase (LPL) deficiency

4. T Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years

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