Adeno-associated virus vectors for gene therapy: more pros than cons?
PE Monahan, RJ Samulski - Molecular medicine today, 2000 - cell.com
PE Monahan, RJ Samulski
Molecular medicine today, 2000•cell.comGene therapy vectors based on the adeno-associated virus (AAV) are being developed for a
widening variety of therapeutic applications. Enthusiasm for AAV is due, not only to the
relative safety of these vectors, but also to advances in understanding of the unique biology
of this virus. This review examines a number of long-standing concerns regarding the utility
of AAV for gene transfer in light of many new insights into the biology, immunology and
production of AAV.
widening variety of therapeutic applications. Enthusiasm for AAV is due, not only to the
relative safety of these vectors, but also to advances in understanding of the unique biology
of this virus. This review examines a number of long-standing concerns regarding the utility
of AAV for gene transfer in light of many new insights into the biology, immunology and
production of AAV.
Abstract
Gene therapy vectors based on the adeno-associated virus (AAV) are being developed for a widening variety of therapeutic applications. Enthusiasm for AAV is due, not only to the relative safety of these vectors, but also to advances in understanding of the unique biology of this virus. This review examines a number of long-standing concerns regarding the utility of AAV for gene transfer in light of many new insights into the biology, immunology and production of AAV.
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