This series is for genetics health professionals led by the Canadian College of Medical Genetics and co-sponsored by Can-GARD. Speakers will be translation-focused researchers, clinicians and innovators. The series will help attendees anticipate, access, and apply new approaches and technologies for patient care.
The Can-GARD/CCMG Leading Strand Series is a self-approved group learning activity (Section 1) as defined by the Maintenance of Certification Program of the Royal College of Physicians and Surgeons of Canada.
To register, click the button below or go to https://us06web.zoom.us/webinar/register/WN_MzE5qgCSQCyPI-X9Ys7_IQ
2024-2025 Leading Strand from 12:00pm – 1:00pm ET, scheduled dates are:
Friday September 27, 2024
Friday October 25, 2024
Friday November 22, 2024
Friday December 13, 2024 – cancelled
Friday, January 31, 2025
Liver-Directed Gene Therapy for Inherited Metabolic Disorders
Learning objectives:
- Quote the most commonly used vectors for liver-directed gene therapy.
- Assess the safety and efficacy of gene therapy, especially for inherited metabolic diseases.
- Understand the current limitations and issues of liver-directed gene therapy.
Description of talk:
Inherited metabolic diseases (IMD) are disorders affecting human biochemical pathways and represent attractive targets for gene therapy because of their severity, high overall prevalence, lack of effective treatments, and possibility of early diagnosis through newborn screening. The liver is a central organ involved in several metabolic reactions and is a favorite target for gene therapy in many IMD. Adeno-associated virus (AAV) vectors have emerged in the last years as the preferred vectors for in vivo gene delivery. Gene replacement strategies are aimed either at correcting liver disease or providing a source for production and secretion of the lacking enzyme for cross-correction of other tissues. A number of preclinical studies have been conducted in the last two decades and, for several diseases, gene therapy has reached the clinical stage. In my talk, our experience on two ongoing clinical gene therapy trials for IMD and I will highlight current obstacles and future strategies to overcome them.
Speaker: Dr. Nicola Brunetti
Bio:
Nicola Brunetti-Pierri graduated in Medicine and trained in Pediatrics at University of Naples Federico II. He then moved to Baylor College of Medicine, Houston, TX for a post-doctoral fellowship and clinical training in Medical Genetics and Biochemical Genetics. He was assistant professor in the Department of Molecular and Human Genetics of Baylor College of Medicine until he returned to Italy. As a physician-scientist his research goals are to elucidate the pathogenetic mechanisms and develop effective therapies for genetic diseases. He identified several new genetic diseases and generated proof-of-concept studies for new therapies in genetic disorders. He received awards from ASGCT and ESGCT and fundings by NIH and ERC. He is the principal investigator of phase 1/2 gene therapy clinical trials. He is Professor of Pediatrics at Federico II University of Naples, Principal Investigator at the Telethon Institute of Genetics and Medicine (TIGEM), Italy.
Friday February 28, 2025
Friday, March 28, 2025
Friday, April 25, 2025
Friday, May 30, 2025
Friday June 27, 2025
Friday, May 16, 2025
Friday, June 20, 2025
