This presentation discusses drug target identification and validation. It introduces drug targets as specific sites where drugs bind to exert their therapeutic effects. Target identification methods include genomics, proteomics, and bioinformatics. Targets are then validated using techniques like siRNAs and antisense oligonucleotides to demonstrate the functional role of targets in disease and ensure drug interactions produce the desired therapeutic response.

1. Presented By-
Snigdha Srivastava
M. Pharm (1st year)
Ram-Eesh Institute of Vocational
& Technical Education

2. ABJURATION
This presentation is made only for
educational purpose only as well as to
give you a general preview about the topic.
Any point ,picture and content mentioned in this
presentation may be modified.
There is no relation of this presentation for their
original documentation.

3. CONTENT
 Introduction
 Drug Target Identification
 Target Identification
Genomics
Proteomics
Bioinformatics
 Target Validation
Si RNA
Antisense Oligonucleotide

4. Introduction
Drug Target
 These are the specific sites where drug gets bind
alter or enhance those specific target site and
shows its required therapeutic action.
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5. Drug Target Types/ Diversity of Drug
Targets
 These are cellular or molecular structures which are
responsible for pathological activity.
 These are proteinitious in nature
 These are following types-
1) Receptor
2)Enzymes
3) Nucleic Acid
4) Ion Channel

6. Target Identification
 Identification of the possible therapeutic target and its
role in the disease.
 One of the most important step in developing anew drug.
 It is the foremost step for improving efficacy of a new
medical candidate.
Target Identification Strategies
These are of two types -:
a) Target De convolution
b) Target Discovery
1)Target De-convolution-:
 It is a phenotypic approach to drug discovery falls in the
target de-convolution.
 It can be achieved by some mentioned methods-
a) By affinity chromatography
b)Expression cloning protein microarray
c)Reverse transfected cell microarray
d) Biochemical suppression

7. Target Discovery:-
 Target based drug discovery, biological(drug) targets
are already established before the lead discovery starts.
 It can be expoilt numerous approaches including-
a)Crystallography
b)Computational Modelling
c)Genomics Biochemistry
d)Binding Kinetics
1-Genomics-
Target Identification based on genetic or genome
methods leverages the relative ease of working with DNA and
RNA to perform large-scale modifications and measurements.
These methods often use the principle of genetic interaction,
relying on the idea of genetic modifiers (enhancers or
suppressors) to generate target hypotheses.

8. 2-Proteomics-
Proteomics is a scientific discipline concerned with
systematic analysis of proteins present in cells at a given
time under given conditions.
Proteomics includes the identification, characterization and
quantitation of the entire complement of proteins in cells,
tissues or whole organisms with a view to understanding
their function in relation to the life of the cell.
https://www.researchgate.net/profile/Henry_Adeola/publication/314117229/figure/fig2/AS:476165300330496@1490538062095/Role-of-proteomics-
in-personalized-medicine-of-prostate-cancer-Various-proteomics.png

9. 3-Bioinformatics-
It’s a computational techniques for solving biological
problems data problems: representation (graphics), storage and
retrieval (databases), analysis (statistics, artificial intelligence,
optimization, etc.) biology problems: sequence analysis,
structure or function prediction, data mining, etc.
https://www.researchgate.net/profile/Wellison_Jarles_Diniz/publication/315318890/figure/download/fig1/AS:473090380570624@1489804
944391/Some-of-the-bioinformatics-applications-Figure-modified-from-Verli-2014.png

10. Target
Validation
 Target validation is a process of demonstrating the
functional role of identified target in disease.
 Target validation confirms that interaction with target
produce the desired change in behaviour of diseased cells.
(GrahamPatrick)
“Only real validation is if a drug turns out to be safe
and efficacious in a patient “
 A major challenge is that many targets fail late in clinic
several years after initiation of the project.
 Several different target validation techniques can be used
to elucidate target function.
1-siRNAs
2-Antisense

11. ANTISENSE OLIGONUCLEOTIDE
Antisense technology prevent the synthesis of
specific protein.
These nucleotides which are complementary to
their target mRNA.
When these AS-ON combined with target mRNA,
a DNA/RNA hybrid form which degraded by the
enzyme RNAs H.
RNAs H is a non specific endonuclease which
catalyse cleavage of RNA.

12. siRNAs
 Small (or short) interfering RNA (si RNA) is the
commonly used for RNA interference (RNAi) tool for
inducing short-term silencing of protein coding genes.
 It is a double stranded RNA molecule which interferes
with the expression of specific genes by degrading mRNA
after transcription & preventing translation.
 si RNA is double stranded RNA(ds RNA).
 It consist of two RNA strands, an antisense (or guide)
strand and a sense (or passenger) strand, which form a
duplex 20-24 bp length.