2. TEAM MEMBERS:
• YASHIKA KORANGA
• AVANTIKA
• KOMAL
• MANSI TYAGI
• SHEFALI
AMITY INSTITUTE OF
MOLECULAR MEDICINE AND
STEM CELL RESEARCH
9/3/20XX Presentation Title 2
3. GENE THERAPY: INTRODUCTION
• Process that alters the biological
properties of a living cell for
therapeutic use.
• Manipulates the expression of
DNA(gene).
• Gene therapy products are being
studied to treat diseases
including cancer, genetic
diseases, infectious diseases
,etc.
9/3/20XX Presentation Title 3
4. GENE THERAPY: TYPES
1. SOMATIC GENE THERAPY
• Occurs in somatic cells.
• Therapeutic genes are incorporated into
somatic/stem cells of the body.
• Safest and best method for gene
therapy.
2. GERMLINE GENE THERAPY
• Occurs in germ cells.
• Adopted to treat genetic diseases.
• Has legal issues.
9/3/20XX Presentation Title 4
5. GENE THERAPY: METHODS
1. IN-VIVO GENE THERAPY
• Direct delivery of genetic material.
• Works with help of a vector.
2. EX-VIVO GENE THERAPY
• Process of removing specific cells from
person, genetic alteration and
replacement.
• Mostly applied to hematopoietic stem
cells.
9/3/20XX Presentation Title 5
6. GENE THERAPY
ADVANTAGES
• Has potential to eliminate and prevent
hereditary diseases.
• Can ‘silence’ a gene.
• Can be used to eradicate a disease from a
person and their future offspring.
DISADVANTAGES
• Short-lived nature.
• Immune response reduces gene therapy
effectiveness.
• Multigene disorders are difficult to treat.
• Side effects.
• Abortions.
9/3/20XX Presentation Title 6
7. ETHICAL ISSUES
• Manipulation of living organisms by human race have to go through rules and regulations.
• Ethical standards are required.
• Raises questions like:
a) How can “good” and “bad” uses of these technologies be distinguished?
b) Who decides which traits are normal and which constitute a disability or disorder?
c) Will the high costs of gene therapy make it available only to the wealthy?
d) Could the widespread use of gene therapy make society less accepting of people who
are different?
e) Should people be allowed to use gene therapy to enhance basic human traits such as
height, intelligence, or athletic ability?
9/3/20XX Presentation Title 7
