Gene Therapy Progress and Prospects : Development of improved lentiviral and retroviral vectors - design, biosafety, and production

P. L SINN; S. L SAUTER; P. B MCCRAY

doi:10.1038/sj.gt.3302570

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Gene Therapy Progress and Prospects : Development of improved lentiviral and retroviral vectors - design, biosafety, and production

Journal article

Peer reviewed

Gene Therapy Progress and Prospects : Development of improved lentiviral and retroviral vectors - design, biosafety, and production

P. L SINN, S. L SAUTER and P. B MCCRAY

Gene therapy, Vol.12(14), pp.1089-1098

2005

DOI: 10.1038/sj.gt.3302570

PMID: 16003340

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Abstract

Replication defective vectors derived from simple retroviruses or the more complex genomes of lentiviruses continue to offer the advantages of long-term expression, cell and tissue specific tropism, and large packaging capacity for the delivery of therapeutic genes. The occurrence of adverse events caused by insertional mutagenesis in three patients in a gene therapy trial for X-linked SCID emphasizes the potential for problems in translating this approach to the clinic. Several genome-wide studies of retroviral integration are now providing novel insights into the integration site preferences of different vector classes. We review recent developments in vector design, integration, biosafety, and production.

Biotechnology

Gene Therapy

Fundamental and applied biological sciences. Psychology

Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy

Health. Pharmaceutical industry

Industrial applications and implications. Economical aspects

Transfusions. Complications. Transfusion reactions. Cell and gene therapy

Applied cell therapy and gene therapy

Biological and medical sciences

Medical sciences

Details

Title: Subtitle: Gene Therapy Progress and Prospects : Development of improved lentiviral and retroviral vectors - design, biosafety, and production
Creators: P. L SINN - Program in Gene Therapy, Department of Pediatrics, University of Iowa Carver College of Medicine, Iowa City, IA, United States
S. L SAUTER - Virogenics Inc, San Diego, CA, United States
P. B MCCRAY - Program in Gene Therapy, Department of Pediatrics, University of Iowa Carver College of Medicine, Iowa City, IA, United States
Resource Type: Journal article
Publication Details: Gene therapy, Vol.12(14), pp.1089-1098
Publisher: Nature Publishing Group
DOI: 10.1038/sj.gt.3302570
PMID: 16003340
ISSN: 0969-7128
eISSN: 1476-5462
Language: English
Date published: 2005
Academic Unit: Microbiology and Immunology; Pulmonary Medicine; Stead Family Department of Pediatrics; Internal Medicine
Record Identifier: 9984093365302771

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