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The document provides an overview of gene therapy, including its definition, history, types, vectors, success cases, advantages, disadvantages, and ethical issues. It discusses the potential of gene therapy to treat genetic diseases and highlights significant milestones in its development. The conclusion emphasizes the complexities and accessibility challenges of gene therapy, while also noting its revolutionary potential in medicine.

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14 views17 pages

Document 1

The document provides an overview of gene therapy, including its definition, history, types, vectors, success cases, advantages, disadvantages, and ethical issues. It discusses the potential of gene therapy to treat genetic diseases and highlights significant milestones in its development. The conclusion emphasizes the complexities and accessibility challenges of gene therapy, while also noting its revolutionary potential in medicine.

Uploaded by

scar.zz4680
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© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
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INDEX

 ACKNOWLEDGEMENT
 INTRODUCTION
 HISTORY
 TYPES OF GENE THERAPY
 VECTORS IN GENE THERAPY
 SUCCESS CASES
 PROS AND CONS
 CONCLUSION
 BIBLIOGRAPHY
INTRODUCTION

WHAT IS GENE THERAPY ?

An approach of treating
diseases by either
modifying the
expressions of an
individual’s genes or
correction of abnormal
genes.
 Replacing a mutated gene that
causes disease with a healthy copy of
the gene.
 Introducing a new gene into the
body to help fight a disease.
HISTORY

 1960’s: The concepts of Gene


Therapy were introduced.
 1990: The first approved gene
therapy in the US took place on 14
September 1990, at the National
Institutes of Health (NIH), under the
direction of William French Anderson.
 2006: successful use of gene therapy
to treat two adult patients for X-linked
chronic granulomatous disease.
 2007: first gene therapy trial for
inherited retinal disease.
TYPES OF GENE
THERAPY

 GERM LINE GENE THERAPY


 SOMATIC GENE THERAPY
Types of somatic cell gene therapy :

 In vivo - Genes are changed in cells


when the cells are still in the body,
called in vivo because the gene is
transferred to cells inside the patient’s
body.
 Ex vivo - C ells are modified outside
the body and then transplanted back in
again, called ex vivo because the cells
are treated outside the body.
EXAMPLES:

1. In vivo –
 In patients with cystic fibrosis, a
protein called cystic fibrosis trans-
membrane regulator (CFTR) is absent
due to a gene defect.
 In the absence of CFTR chloride
ions concentrate within the cells and it
draws water from surrounding.
 This leads to the accumulation of
sticky mucous in respiratory tract and
lungs.
 Treated by in vivo replacement of
defective gene by adenovirus vector .
2. Ex vivo –
 1st gene therapy – to correct
deficiency of enzyme, Adenosine
deaminase (ADA).
 SCID- Severe Combined
Immunodeficiency caused due to
defect in gene coding for ADA.
 Deoxyadenosines accumulate and
destroys T lymphocytes. Disrupts
immunity.
VECTORS

 To transfer the desired gene into a


target cell, a carrier is required. Such
vehicles of gene delivery are known
as vectors.
 2 main classes
1.Viral vectors
2.Non-viral vectors

VIRAL VECTORS :
 Viruses introduce their genetic
material into the host cell as part of
their replication cycle.
 remove the viral DNA and using the
virus as a vehicle to deliver the
therapeutic DNA.
 The viruses used are altered to make
them safe, although some risks still
exist with gene therapy.

Types of viral vectors:


 Several viruses have been used for
human gene therapy, including :
 Retrovirus
 Adenovirus
 Adeno-associated virus
 Herpes simplex virus
NON-VIRAL VECTOR :
 Direct introduction of pure DNA
construct into target tissue.
 Poly-L-lysine bound to specific target
cell receptor; Therapeutic DNA is then
made to combine with the conjugate
to form a complex.
 DNA construct surrounded by
artificial lipid layer, most of it gets
degraded by lysosomes.
SUCCESS CASES

 Gene therapy cure blindness of


inherited condition.
 Gene therapy reduces Parkinson’s
diseases symptoms - It has
significantly improved the weakness
of the symptoms such as tremors,
motor skill problems, and rigidity.
PROS AND CONS

ADVANTAGES :
 Gene therapy has the potential to
eliminate and prevent hereditary
diseases such as cystic fibrosis, ADA-
SCID etc.
 It is a possible cure for heart disease,
AIDS and cancer.
 It gives someone born with a genetic
disease a chance to life.
 It can be used to eradicate diseases
from the future generations.

DISADVANTAGES :
 Long lasting therapy is not achieved
by gene therapy; Due to rapid
dividing of cells benefits of gene
therapy is short lived.
 Immune response to the transferred
gene stimulates a potential risk to
gene therapy.
 Disorders caused by defects in
multiple genes cannot be treated
effectively using gene therapy.
ETHICAL ISSUES :
 Who will have access to therapy?
 Is it interfering with God’s plan?
 Should people be allowed to use gene
therapy to enhance basic human traits
such as height, intelligence etc.?
 Is it alright to use the therapy in the
prenatal stage of development in
babies?
CONCLUSION

 Theoretically, gene therapy is the


permanent solution for genetic
diseases.
 But it has several complexities. At its
current stage, it is not accessible to
most people due to its huge cost.
 A breakthrough may come anytime
and a day may come when almost
every disease will have a gene therapy
 Gene therapy have the potential to
revolutionize the practice of medicine.
BIBLIOGRAPHY

1. https://www.genome.gov/genetics-
glossary/Gene-Therapy

2. https://www.fda.gov/vaccines-blood-
biologics/cellular-gene-therapy-products/
what-gene-therapy

3. https://www.google.com/

4. NCERT TEXTBOOK CLASS XII


BIOLOGY

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