Gene Therapy. What is gene therapy? Its an experimental science used to help treat and prevent diseases by introducing DNA/RNA into a persons cells. Approaches.
Gene therapy
SRC lunch speak talk 150323 - PSJ intro
ZFNs: Zinc finger chimeric endonucleases -- a way to make a site-specific double-stranded break at a chosen site Link zinc fingers to a nonspecific endonuclease.
Outcomes: MDA-NINDS Workshop on Best Practices for Gene Therapy Programs (April 2014) (…and NINDS’ Adaptations) John D. Porter, Ph.D. Program Director.
DNA TECHNOLOGY Developments and Applications. Genetic Engineering Modifying an organism’s genotype by introducing genes that have never been present in.
Link zinc fingers to a nonspecific endonuclease (a dimer).
Gene therapy for Leber congenital amaurosis (LCA) caused by damage to the RPE65 gene
10 Medical Biotechnology
10 Medical Biotechnology I. Gene Therapy A. Genetic Disorders B. Gene Target Selection C. Gene Delivery Methods D. Viral vectors 1. Retrovirus2. Adenovirus3.
