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Gene Therapy

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40 views26 pages

Gene Therapy

Uploaded by

deseseamor829
Copyright
© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
Available Formats
Download as PPTX, PDF, TXT or read online on Scribd
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Gene

Therapy
Gene therapy is a
medical
technique aimed
at treating or
preventing
diseases by
correcting or
altering genes
inside the
Modifying or
replacing faulty
genes with
functional ones to
restore normal
function.
1972: Gene therapy
concept introduced.

1990: First successful


human trial to treat a
genetic disorder
(adenosine deaminase
deficiency).
Applications Today

Treats single-
gene disorders Emerging
like cystic therapies for
fibrosis and cancer, HIV, and
hemophilia. degenerative
diseases.
Two Types of Gene
Therapy
1. Somatic Gene Therapy
Alters non-reproductive cells (e.g., liver, muscle,
or blood cells).
Examples: Treating hemophilia or muscular
dystrophy.
Changes do not pass to the next generation.
Two Types of Gene
Therapy
2. Germline Gene Therapy
Modifies sperm or egg cells (reproductive cells).
Advantages: Can eliminate hereditary diseases.
Controversy: Ethical concerns over long-term effects
and "designer babies."
Changes are inherited by future generations.
Stem Cell Gene Therapy
- Found in bone marrow, umbilical cord blood, or
induced in labs (iPSCs).
Role in Gene Therapy:
Delivery Mechanism: Stem cells engineered with
therapeutic genes can target specific tissues.
Tissue Regeneration: Stem cells can replace damaged or
diseased tissue with genetically corrected cells.
Stem Cell Gene Therapy
Applications:
Cancer Treatment: CAR-T cell therapy for blood cancers.
Genetic Disorders: Correcting blood diseases like sickle
cell anemia or thalassemia.
Advantages of Stem Cell Therapy:
Long-lasting and precise results.
Potential to cure diseases at their source
Bioethics of Gene
1. Safety Concerns
Therapy
Risk of unintended mutations or "off-target"
effects.
Long-term effects on health are still unknown.
Example: Immune system reaction to viral vectors
in earlier trials.
Bioethics of Gene
Therapy
2. Accessibility
High costs of gene therapy limit
availability to wealthy individuals or
nations.
Need for equitable distribution of
advanced treatments.
Bioethics of Gene
Therapy
3. Germline Gene Editing Debate
Pros:
Could eliminate genetic disorders from the population.
Offers permanent solutions.
Cons:
Opens the door to non-medical enhancements (e.g., intelligence,
appearance).
Ethical implications for altering future generations without consent.
Bioethics of Gene
Therapy
4. Informed Consent
Ensuring patients and families fully
understand risks and benefits.
Controversy in cases involving children or
incapacitated individuals.
Real-
Life
Case
Studies
SMA Treatment (Spinal
Muscular Atrophy):

Zolgensma, a gene
therapy, has saved lives of
children with this fatal
condition.
CAR-T Therapy:

Revolutionized cancer
treatment by
engineering T-cells to
target tumors.
Challenges and Future
Directions
Current Limitations:
●High costs: Average treatment
costs exceed $1 million.
●Complexity of delivery
methods.
●Limited success in treating
multi-gene disorders.
Challenges and Future
Directions
Future Trends:
Advances in CRISPR and base editing
for precision.

Development of non-viral delivery


systems.

Broader clinical trials for rare and


common diseases.
Multiple
Sclerosis
Problem with
his visions
Lung
Muscle infection
tear Knee injury
Bioethics: Bioethics is the study
of ethical issues emerging from
advances in biology and
medicine
The ethical questions surrounding gene therapy and genome
editing include:
● How can “good” and “bad” uses of these technologies be
distinguished?
● Who decides which traits are normal and which constitute a
disability or disorder?
● Will the high costs of gene therapy make it available only to the
wealthy?
● Could the widespread use of gene therapy make society less
accepting of people who are
different?
● Should people be allowed to use gene therapy to enhance basic
human traits such as height, intelligence, or athletic ability?

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