3. Antigene and Antisense Therapy
• Blocking transcription using a single stranded nucleotide sequence (
antigene oligonucleotide) that hybridizes with specific gene, this is
called as Antigene therapy.
• Antisense Therapy refers to the Inhibition of translation by using a
single stranded nucleotide ( Antisense oligonucleotide)
• It also possible to inhibit transcription and translation by blocking
with oligonucleaotide the transcription factor responsible for the
specific gene expression.
4. • Nucleic acid therapy refers to use of DNA or RNA molecules for
therapeutic purpose,
5.
6.
7.
8. Antisense therapy for Cancer
• Oncogene :
• Antisense oligonucleotide are use for the treatment of myeloid
leukemia in 1991.
• This approach is effective only if the antisense oligonucleotide
specifically bind to target mRNA and block protein biosynthesis.
• This can achieved in two ways illustrated in Next slide.
•
9.
10. Anti sense therapy for HIV
The target cell for HIV infection are genetically
engineered to contain a gene that can express a
complementary copy of HIV genome.
The gene produces antisense RNA
Cell containing antisense RNA infected by HIV
Antisense RNA bind to viral genome (RNA)
For RNA-RNA hybrid molecules
Double stranded molecules cannot be used by
enzymes Reverse transcriptase
11.
12.
13. Advantages of Gene therapy
• It is permanent solution for genetic diseases.
• It is possible to cure Cancer, HIV
• It also helps to eradicated genetically related disease
14. limitations
• Due to some limitation gene therapy has not progressed the way it
should ,despite intensive research.
• Break though may come anytime and of course this is only possible
with the persistent research.
• It might take some year.