Gene Therapy New by vishal meena mbbs.pptx

Gene therapy

1. Presented By: Dr. Sanjay Bhatt, Assistant Professor Dept. of Biochemistry, MDB ASMC Deoria , UP Gene Therapy

2. What is gene therapy? Gene therapy is a medical approach that treats or prevents disease by correcting the underlying genetic problem . Gene therapy techniques allow doctors to treat a disorder by altering a person’s genetic makeup instead of using drugs or surgery. The earliest method of gene therapy, often called gene transfer or gene addition , was developed to: • Introduce a new gene into cells to help fight a disease. • Introduce a non-faulty copy of a gene to stand in for the altered copy causing disease.

3. Gene Therapy When gene therapy is when DNA is introduced into a patient to treat a genetic diseases. New gene=Functioning gene Faulty gene Functioning gene Gene Therapy

4. History GT initially started by Martin cline in 1980. Between 1989 – 2018= 2900 clinical trial which has been done. Spark Therapeutics Novartis Private organization performed GT process and approved by FDA in 2017 Novartis Zolgensama Alnylam’s Patisiran Drugs (two drugs introduced and approved by FDA in 2017. First approved gene therapy experiment 14 september 1990 US. On a patient ASHANTI DESILVA , she is 4 years old =ADA-SCID (Adenosine deaminase severe combined immuno defficiency )

5. Types GT

6. Types of GT Somatic cell GT: Therapeutic gene transferred into somatic cells. Eg . Bonemarrow cells, Blood cells, skin cells etc. Will not be inherited later generations. 2. Germline GT: Therapeutic gene transferred into the germ cells. eg . Egg , sperm Will be inherited later generations.

7. Approches in GT

8. In Vivo GT In vivo GT Direct delivery of the therapeutic gene into the target cell into patients body. Viral or non viral vectors systems. eg . Cystic fibrosis , CFTR gene Accumulation of sticky mucous in respiratory tract and lungs. Adinovirus vector

9. 2. Ex Vivo GT Bacterium carrying plasmid with cloned normal human ADA gene . eg . ADA-SCID Ex Vivo GT

10. Ex Vivo

11. Vectors Viral Vectors: Retrovirus vector systems Adenovirus System Adeno associated virus vector Herpex simplex virus vector

12. Vectors 2. Non viral vector system Pure DNA construct Lipoplexes DNA molecular conjugates Human artificial chromosomes

13. Tools/methods Gene gun Microinjection Using detergents lipofection