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THE PHARMA REVIEW
(OCTOBER 2008) |
Gene Therapy for Inherited
and Acquired Disorders: A Brief Review
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Pavan Malhotra, O.P. Gupta,
Archana Parihar, Shakti Bala, Geetika Gupta |
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Abstract: Certain diseases are inherited while
some are acquired due to heritable or acquired
abnormalities at the level of gene in the genome of
individuals. Gene therapy refers to the correction or
inactivation of the faulty gene or introduction of a
functional gene to make up for genetic deficiency. The
functional gene required for the transfer is obtained by
recombinant DNA technology. Transfer of gene is carried
out either directly or through a vector. Clinical
application of gene therapy is being tried both in
experimental animals and human beings. More than 300
clinical trials, for gene transfer in patients suffering
from various disorders have been taken up. However gene
therapy is still at an investigational stage. It is
hoped that in the near future, gene therapy will prove
to be an effective modality for treating many inherited
and acquired disorders.
Introduction
Genes are DNA strands. A gene when it expresses, leads
to formation of specific mRNA which in turn leads to
synthesis of specific proteins and subsequently the
specific functions in the body system are performed.
Obviously, a faulty gene will lead to absence or reduced
formation of a protein and/or of its function as it is
likely to express faulty protein and lead to some
acquired disorders like immunological disorders
including AIDS, cardiovascular, neurological, metabolic
or malignancy. While advances made in molecular and
cellular biology describe the proteins that are
mediating the biological functions or disease processes,
the DNA technology provides access to the genes that
control these events and it is now possible to even
define exactly which DNA base pair is anomalous.
Accordingly, different strategies have been developed to
rectify these anomalies at gene level.
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