Gene therapy: promises and problems

A Pfeifer, IM Verma - Annual review of genomics and human …, 2001 - annualreviews.org
Annual review of genomics and human genetics, 2001annualreviews.org
Gene therapy can be broadly defined as the transfer of genetic material to cure a disease or
at least to improve the clinical status of a patient. One of the basic concepts of gene therapy
is to transform viruses into genetic shuttles, which will deliver the gene of interest into the
target cells. Based on the nature of the viral genome, these gene therapy vectors can be
divided into RNA and DNA viral vectors. The majority of RNA virus-based vectors have been
derived from simple retroviruses like murine leukemia virus. A major shortcoming of these …
Gene therapy can be broadly defined as the transfer of genetic material to cure a disease or at least to improve the clinical status of a patient. One of the basic concepts of gene therapy is to transform viruses into genetic shuttles, which will deliver the gene of interest into the target cells. Based on the nature of the viral genome, these gene therapy vectors can be divided into RNA and DNA viral vectors. The majority of RNA virus-based vectors have been derived from simple retroviruses like murine leukemia virus. A major shortcoming of these vectors is that they are not able to transduce nondividing cells. This problem may be overcome by the use of novel retroviral vectors derived from lentiviruses, such as human immunodeficiency virus (HIV). The most commonly used DNA virus vectors are based on adenoviruses and adeno-associated viruses. Although the available vector systems are able to deliver genes in vivo into cells, the ideal delivery vehicle has not been found. Thus, the present viral vectors should be used only with great caution in human beings and further progress in vector development is necessary.
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