Gene Therapy PDF - Faiza Amin - Treatments, Strategies & Methods

2/12/2025 GENE THERAPY BHMS (SEMESTER 4TH) PREPARED BY FAIZA AMIN HISTORY:- On September 14,1990 , W. French Anderson and his collegue at NH performed the first approved gene therapy procedure on a four year girl Ashanthi de silva born with SCID (seve...

2/12/2025 GENE THERAPY BHMS (SEMESTER 4TH) PREPARED BY FAIZA AMIN HISTORY:- On September 14,1990 , W. French Anderson and his collegue at NH performed the first approved gene therapy procedure on a four year girl Ashanthi de silva born with SCID (severe combined immune deficiency) due to lack of enzyme adenosine deaminase (ADA). DEFINITION:- It is technique in which defective gene is replaced by the functional gene so that the body can make the functional protein and therefore eliminate the root cause of disease. AIM OF GENE THERAPY:- i. Replace faulty /defective gene ii. Silencing of faulty gene iii. Introduce new normal gene 1 2/12/2025 2  2/12/2025 GENE DELIVERY SYSTEM:- GENE TRANSFER USING VIRAL VECTOR:- Vector:- A cerrier molecule called vector must be used to deliver the therputic gene to patients target cells. virus bind to their host and introduce their genetic material into host cell. A viral vector is a virus which has been modified in a labortary environment for purpose of introducing genetic material into cell. to form a viral vector remove the gene in virus that cause disease.then replace those gene with genes encoding Method of the desired effect (for instance insulin production in case of diabetes. gene Delivery Target specific gene Virus mediated Direct or non- Good at delivering genetic material transfer viral transfer Some integrate into host genome Can be modified 3  2/12/2025 NON-VIRAL VECTOR SYSTEM:- i. Electroporation ii. Gene gun or biolistic or microprojectile bombardment iii. Microinjection 4 2/12/2025 5