ANTISENSE THERAPY AND ITS UNIQUE APPLICATION TO TREAT GENETIC DISORDERS-A REVIEW"
International Journal of Emerging Technologies and Innovative Research (www.jetir.org), ISSN:2349-5162, Vol.6, Issue 4, page no.194-200, April-2019, Available :http://www.jetir.org/papers/JETIR1904F35.pdf
Authors: Ganesh S. Waghule.
Abstract:
A genetic disorder is a genetic problem
caused by one or more abnormalities formed in the genome. Most genetic
disorders are quite rare and affect one person in every several
thousands or millions. Genetic disorders may be hereditary, meaning that
they are passed down from the parents' genes. In other genetic
disorders, defects may be caused by new mutations or changes to the DNA.
The principle behind the technology is that an antisense nucleic acid
sequence base pairs with its complementary sense RNA strand and prevents
it from being translated into a protein or the inhibition of RNA
processing yields the therapeutic result.The Oligonucleotide are used to
block coded massage carried by mRNA this known as antisense therapy the
primary sequence of mRNA called oligonucleotide contains nucleic acid
bases complimentry to mRNA molecule. An oligonucleotide has a
complimentry base sequence called antisense oligonucleotide, when mixed
with mRNA antisense oligonucleotide Recognize mRNA and forms duplex and
act as barrier to translation process and block protein synthesis.
Antisense drugs have the potential therapeutic efficacy in the treatment
of various diseases than the conventional drug. The rapid development
of antisense technology offers almost unlimited scope for the
development of new and highly specific therapeutics. Antisense
therapeutics therefore is well positioned to play a significant role in
the progression of antisense technology for drug development in human
diseases.
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