Research Journal of Pharmacology and Pharmacodynamics

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Review Article on Gene Therapy


Affiliations
1 Modern College of Pharmacy, Sector 21, Yamunanagar, Nigdi, Pune-44 (M.S), India
2 Sharad Pawar College of Pharmacy, Nagpur, India
     

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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 transducer 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. An example of gene-knockout mediated gene therapy is the knockout of the human CCR5 gene in T-cells in order to control HIV infection. 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.

Keywords

Lentiviruses, Transducer, RNA Vector, Adeno-Associated Viruses.
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  • Review Article on Gene Therapy

Abstract Views: 250  |  PDF Views: 2

Authors

P. M. Patil
Modern College of Pharmacy, Sector 21, Yamunanagar, Nigdi, Pune-44 (M.S), India
P. D. Chaudhari
Modern College of Pharmacy, Sector 21, Yamunanagar, Nigdi, Pune-44 (M.S), India
Megha Sahu
Modern College of Pharmacy, Sector 21, Yamunanagar, Nigdi, Pune-44 (M.S), India
N. J. Duragkar
Sharad Pawar College of Pharmacy, Nagpur, India

Abstract


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 transducer 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. An example of gene-knockout mediated gene therapy is the knockout of the human CCR5 gene in T-cells in order to control HIV infection. 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.

Keywords


Lentiviruses, Transducer, RNA Vector, Adeno-Associated Viruses.

References