This presentation will cover mainly Gene Therapy and their subtopics, This presentation is made by Miss Nadia Muzaffar.

1. Gene Therapy
Presenter
Nadia Muzaffar
Sindh Biotechnologists Association (SBA)
Presenter:
Nadia Muzaffar
Mphil Biotechnology
Sindh Biotechnologists association

2. What is Gene therapy
Replacing a mutated
gene that causes
disease with a
healthy copy of the
gene.
Inactivating, or
“knocking out,” a
mutated gene that is
functioning
improperly.
Introducing a new
gene into the body
to help fight a
disease.

3. Gene?

4. Mutation?
Changes to DNA are called mutations
Changes mRNA
Changes protein
May change trait

6. Genetic disorders
• Sickle cell anemia
• Cystic fibrosis
Single gene
disorders
• Down syndrome
• Cri du chat syndrome
Chromosomal
disorders
• Colon cancer
• Parkinson disease
Complex
disorders

7. What is gene therapy vector?
•DNA based Adenoviruses, Herpes simplex
•RNA based Retroviruses
Viral
vectors
•Injection of naked DNA
•Physical approach Electroporation, gene gun
•Chemical approach Liposome, Polymers,
lipopolyplex, dendrimers,
inorganic nanoparticles
Non viral
vectors

8. Viral Vectors

9. Non-viral Vectors

10. In vivo treatment
Ex vivo
treatment

11. Applications
Treatment of
monogenic
disorders
In future
treatment of
cancer, AIDS
and infectious
disease
Eliminates or
prevents
hereditary
disease
Possible cure
of inborn
genetic
diseases
Limitations
Short-lived
nature
Immune
response of
patient
Vector
toxicity
Multiple
gene
disorders can
not be
treated

12. Picture Links
• https://ghr.nlm.nih.gov/primer/mutationsanddisorders/genemutation
• https://www.utoronto.ca/entrepreneurs/deep-genomics
• https://en.wikipedia.org/wiki/Gene
• https://theglobalscientist.com/2016/03/14/knowledge-is-power-when-genes-predict-cancer-risk-
and-inform-medical-management/
• https://ghr.nlm.nih.gov/primer/basics/gene
• https://medlineplus.gov/geneticdisorders.html
• https://ghr.nlm.nih.gov/primer/mutationsanddisorders/genemutation
• https://ghr.nlm.nih.gov/primer/therapy/procedures
• http://wuhsag.weebly.com/uploads/1/4/0/9/14095127/protein_regulation___mutations.pdf
• https://www.singerinstruments.com/resource/what-are-genetic-mutation/
• https://genetherapy.isth.org/getting-to-know-gene-therapy-terminology-and-concepts-
interactive-module
• https://cellculturedish.com/key-considerations-for-gene-therapy-commercialization/
• https://www.bing.com/images/search?view