Overview / Abstract: |
The goal of gene therapy is to treat diseases at the genetic level by replacing deficient enzymes in target tissues, therefore, changing the course of the disease. Simply stated, it is the transfer of genetic material for disease therapy or prevention. Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-term efficacy in several trials across target organs, including eye, liver, skeletal muscle, and the central nervous system. The goal of this CME-certified activity is to increase physicians' knowledge of clinical considerations relating to the use of adeno-associated virus (AAV) vectors in gene therapy, and its impact on the treatment of several rare disorders. |
Expiration |
Sep 08, 2022 |
Discipline(s) |
Nurse Practitioner , Nursing CNE, Physician CME, Physician Assistant CME |
Format |
Webinar / Webcast / Video |
Credits / Hours |
.75 |
Accreditation |
ACCME |
Presenters / Authors / Faculty |
Eric T. Rush, MD, FAAP, FACMG |
Activity Specialities / Related Topics |
Genetics, Nephrology, Pathology, Pediatrics |
Sponsors / Supporters / Grant Providers |
Ultragenyx Pharmaceutical |
Keywords / Search Terms |
EXCEL Continuing Education Rare disorders, (please add hepatology to your specialty list), Adeno-Associated Virus, gene therapy. Free CE CME |