BIOLOGICAL ADVANCEMENTS

BEYOND THE IMAGINATION

GENE THERAPY
What is Gene Therapy
 A branch of Regenerative Medicine, an emerging field that
involves the "process of replacing, engineering or
regenerating human cells, tissues or organs to restore or
establish normal function”.

 Gene therapy is the the delivery of therapeutic gene into a

patient's cells to treat disease.
 Different Routes of Gene Therapy

• Ex vivo gene therapy -

• Usually with blood cells (lymphocytes or blood
stem cells) for diseases affecting the hematopoietic
system

• In vivo gene therapy -

• Oncolytic adenoviruses for the treatment of cancer
• Adeno-associated vectors for the treatment of
Duchenne muscular dystrophy or hemophilia
• Non-viral for cancer
Sterilizing vs functional cure
 Sterilizing Cure
 complete eradication of
all replication competent
forms of HIV. The
reservoir is gone.
 Functional Cure
 Life-long control of virus
in the absence of
antiretroviral therapy, but
without achieving
complete eradication of
HIV.Virus remains in
reservoirs in the body.
Gene Therapy in Blood Cells
Some targets for gene therapy
Some targets for gene therapy
Gene Therapy- Vectors to deliver anti-
HIV genes
 LV- Lentivirus vectors
 RV- gammaretroviral
vectors,
 AAV – adeno-
associated vectors
 Adenovirus vectors

 Vectors are replication

defective – so they
cannot replicate and
spread once they are
inside the cells and after
delivering the anti-HIV
genes
Next Generation COOH

Technology Zinc finger

Genome editing
 Zinc finger NH2

 TAL Effector TAL Effector

Nuclease Nuclease

 CRISPR/Cas9 CRISPR/Cas9

 MegaTals megaTAL
Site-Specific Gene Targeting / Engineering

HIV target gene eg CCR5

Thanks to Barry Stoddard

 Scarless Repair Of Genetic Defect or
Targeted Insertion Of New Genetic Material
 Scarless Repair Of Genetic Defect or
Targeted Insertion Of New Genetic Material
 Scarless Repair Of Genetic Defect or
Targeted Insertion Of New Genetic Material
 Scarless Repair Of Genetic Defect or
Targeted Insertion Of New Genetic Material
Conclusions
 Current approaches in trial are very
complex, but as the technologies develop,
easier to administer and cheaper therapies
will be available.
 Risks, such as off-target effects and the need
for treatment interruptions, are high in early
trials and participants should carefully
consider all risks before entering a trial.
“Technology always goes forward.
There are radical new technologies that
surprise us all the time.
And we’ve got a long time in the future to go.
This is our conclusion:
Human evolution will be self-driven.”
• PRESENTED BY-
• JAGTENDRA SINGH INDOLIA
• AMBUJ JAIN
• PRADYUMN MITTAL

THANK YOU