gene_therapy_teacher.pptVIP

GENE THERAPY Diseases for applying gene therapy Gene therapy could be very different for different diseases Gene therapy in vivo and ex vivo schemes Injections of naked DNA Current attempts with naked DNA vaccination in infectious diseases Ballistic DNA Injection (gene guns) Liposomes DNA delivery of genes by liposomes Cystic fibrosis Lungs in cystic fibrosis Cystic fibrosis lungs are prone to infections MOST COMMON VIRAL VECTORS Retroviral vectors are able to infect dividing cells only Real treatments performed with retroviral system What is Severe Combined Immunodoficiency (SCID)? SCID treatments * Disease Defect Target cell Severe combined Bone marrow cells or immunodeficiency T-lymphocytes Hemophilia Liver, muscle Cystic fibrosis Lung Cells Cancer Many cell types Neurological diseases Parkinson’s/ Alzheimers Nerve Cells Infectious diseases AIDS, hepatitis B White Blood Cells Gene transplantation (to patient with gene deletion) Gene correction (To revert specific mutation in the gene of interest) Gene augmentation (to enhance expression of gene of interest) In vivo Ex vivo :8237/M288/SChow_4_10/sld005.htm IN VIVO EX VIVO 1. The genetic material is transferred directly into the body of the patient 2. More or less random process; small ability to control; less manipulations 3. Only available option for tissues that can not be grown in vitro; or if grown cells can not be transferred back In vivo gene therapy The genetic material is first transferred into the cells grown in vitro 2. Controlled process; Genetically altered cells are selected and expanded; more manipulations 3. Cells are then returned back to the patient Ex vivo gene therapy HIV Hepatitis Influenza Tuberculosis, Lyme disease Malaria Invented for DNA transfer to plant cells Fully applicable to eukaryotic cells plasmid DNA shown here Next level id