Rate limiting steps of AAV transduction and implications for human gene therapy

S Sanlioglu; M M Monick; G Luleci; G W Hunninghake; J F Engelhardt

doi:10.2174/1566523013348788

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Rate limiting steps of AAV transduction and implications for human gene therapy

Journal article

Peer reviewed

Rate limiting steps of AAV transduction and implications for human gene therapy

S Sanlioglu, M M Monick, G Luleci, G W Hunninghake and J F Engelhardt

Current gene therapy, Vol.1(2), pp.137-147

07/2001

DOI: 10.2174/1566523013348788

PMID: 12108951

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Abstract

Despite the fact that adeno-associated virus type 2 (AAV2) is an extremely attractive gene therapy vector, its application has been limited to certain tissues such as muscle and the brain. In an attempt to broaden the array of target organs for this vector, molecular studies on the mechanism(s) of AAV transduction have expanded over the past several years. These studies have led to the development of innovative strategies capable of overcoming intracellular barriers to AAV2 transduction. The basis of these technologic breakthroughs has stemmed from a better understanding of the molecular processes that control AAV entry and intracellular trafficking to the nucleus. This review will focus on the identification of molecular components important for recombinant AAV (rAAV) transduction while highlighting the techniques used to discover them and potential clinical application of research findings.

Dependovirus - genetics

Endocytosis

Animals

Transduction, Genetic

Humans

Models, Genetic

Active Transport, Cell Nucleus

Genetic Vectors

Gene Conversion

Genetic Therapy - methods

Details

Title: Subtitle: Rate limiting steps of AAV transduction and implications for human gene therapy
Creators: S Sanlioglu - Department of Internal Medicine, Division of Pulmonary and Critical Care, Center for Gene Therapy, University of Iowa, College of Medicine, Iowa City, IA 52242, USA. sanlioglus@mail.medicine.uiowa.edu
M M Monick
G Luleci
G W Hunninghake
J F Engelhardt
Resource Type: Journal article
Publication Details: Current gene therapy, Vol.1(2), pp.137-147
Publisher: United Arab Emirates
DOI: 10.2174/1566523013348788
PMID: 12108951
ISSN: 1566-5232
eISSN: 1875-5631
Grant note: HL58340 / NHLBI NIH HHS HL-60316 / NHLBI NIH HHS ES-09607 / NIEHS NIH HHS P30 DK54759 / NIDDK NIH HHS
Language: English
Date published: 07/2001
Academic Unit: Roy J. Carver Department of Biomedical Engineering; Pulmonary, Critical Care, and Occupational Medicine; Anatomy and Cell Biology; Radiation Oncology; Internal Medicine
Record Identifier: 9984025432202771

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