antisense technology and new applications

Classification of Antisense therapy. MOA . Application of Antisense therapy

1. ANTISENSE TECHNOLOGY By Chandana H S 2 nd Sem M Pharm Reg no: 23PP051 Shree Devi college of Pharmacy Manglore

2. Introduction Conventional definition of antisense: manipulation and/or modification of DNA or RNA

so that its component (nucleotides) forms a complementary copy of normal or “sense” messenger RNA.

3. What is Antisense technology? The technique in which translation of mRNA into proteins is inhibited by introducing single stranded nucleotide (oligo de oxy nucleotide) • The oligonucleotides are complementary to the mRNA, which physically binds to mRNA.
This technology prevent the synthesis of specific proteins.

4. History First time at “Free University of Amsterdam”, used antisense RNA technology against the gene determining flower color of Petunia. Antisense effect first demonstrated by Zemencnick and Stephenson in 1970 on “ Robus sarcoma virus”.

First time antisense oligonucleotides are Onthesized by Eckstein and colleagues.

5. Antisense nucleic acid sequence +
Specific mRNA Interrupt normal cellular processing of gene

6. Antisense technologies are a suite of techniques Together form a very powerful weapon For studying gene function and for discovering more specific treatments of disease.

7. Antisense Oligonucleotides Oligodeoxynucleotides are 15-20 units nucleotides, which can hybridize with specific target mRNA.

Oligonucleotides can hybridize with different types of RNA

-transcripts-mRNAs

-intron-exons

-double stranded RNAs

8. siRNA • Known as silencing RNA

• Commonly used RNAi (interference)

• Double stranded RNA, 20-25 base pair,

similar to miRNA and operate with R

interference pathway.

9. Application Medical application -In cancer chemotherapy.

- In AIDS therapy. Veterinary application - medicine and pharmaceuticals

- transgenic animals for improved milk production.

10. Agricultural application -genetic application of fruit ripening

-modification of flower color in decorative flower.

• Industrial application -new drug discovery

11. Role In Drug Discovery In recent years, Antisense oligonucleotides (AS-OD) technologies have been widely used as potent and promising tools for drug discovery and development.

Diseases are connected to insufficient or excess production of certain proteins.

• If the production of these proteins are interrupted then many diseases can be cured.

12. The antisense approach could reduce the cost of drug discovery
Major improvements have been achieved by the development of modified nucleotides that provide high target affinity, enhanced biostability and low toxicity.

13. Thank you