PPT-Topic – Type of Gene Therapy

Presented by Ms P H Giri Department of Microbiology Deogiri College Aurangabad Ms Priyanka H Giri BSc T Y Semester VI Paper No XIX Recombinant DNA Technology Unit 4. . Gene therapy: . to correct a . genetic defect . by transferring of a . functional normal copy. of the gene into cells. Examples of diseases caused by genetic defect. Ornithine . transcarbamylase. William Brooks. Medicinal Chemistry. March 31, 2011. Outline. Conventional drug design vs. gene therapy. Examples of genetic engineering. Types of gene therapy. Mechanisms. Delivery vectors. Viral. Non-viral. Cell/Gene Therapy by: . Jeff Sheehy, the California Institute for Regenerative Medicine (CIRM). Jerome Zack, UCLA. Hans-Peter . Kiem. , The Fred Hutchinson Cancer Research Center. Jessica . Handibode. Therapy of enzyme defects: general considerations. How many organs are affected by the enzyme defect: One organ, a few, or all organs?. How severe is the defect?. Can the defect be adequately controlled by conventional treatment?. Genetic Counseling. Treating . Genetic . Disease. Removing . an affected body . part.. Replacing . an affected body part or biochemical . with material . from a . donor.. Delivering . pure, human proteins derived . Dept. of Medical Genetics & Medical Biology. Gene Therapy cartoon 10 - search ID shrn157. . . Gene Therapy cartoon 5 - search ID shrn147. . . Purpose of. gene therapy:. Management and correction of human diseases. Propelled by the promise of faster, cheaper, and more accurate by CRISPR-Cas gene editing. For all of their ease and rapidity, cell or gene therapy. The generation of unintended DNA strand its niche w J Gynecol Oncol Vol. 19, No. 4:251-255, 2008Hong-Bum Cho, et al.252Table 1. Sequences of consensus oligonucleotides used to amplifica-tion on and geneNameSequence (5' to 3')TargetReferencesGP5+GP6+G December 1984 NTIS order #PB85-206076 “Where two principles really do meet which cannot be reconciled with one another, theneach man declares the other a fool and heretic. ” –Ludwig Wit Msc. . . level. College of Pharmacy . Al-. Mustansiriyah. University. Dr. . Basma. Al- . Sudani. Lecture . 2. 9-11-2021. Table. . of Content. What Genes can do. Why Genetic Disorders. Law of Inheritance. Cary O. Harding, MD. Department of Molecular & Medical Genetics. Disclosures. BioMarin Corporation. Funds for participation in clinical trials. Sapropterin dihydrochloride. rAvPAL-PEG. National PKU Alliance. Starter: Comparing DNA processes. Process. What does it do?. How does it work?. PCR (Polymerase Chain. Reaction). DNA Sequencing. DNA Profiling. Gel Electrophoresis. Process. What does it do?. How does it work?. 2020. Monogenic and Complex Diseases. Gene therapy is a promising approach for both monogenic and complex diseases. 1. 1. Wang D, Gau G. . Discov Med . 2014;18:151–161; 2. Ginn SL, et al. . J Gene Med. techniques . Content. Introduction. Importance of Gene transfer. Types of gene transfer techniques. Method. Application. Introduction. Gene transfer is to transfer a gene from one DNA molecule to another DNA molecule..