Tag Archives: clinical trials

Episode 17: Gene Therapy in TSC



In episode 17 of TSC Now, host Dan Klein talks to Xandra Breakefield, PhD, Professor of Neurology at Harvard Medical School and a geneticist, specializing in neurology & tadiology at Massachusetts General Hospital. Dr. Breakefield’s research focuses on gene therapy in preclinical mouse models of TSC in which the TSC1 or TSC2 gene are missing. By introducing the missing gene into the bloodstream via an adeno-associated virus (AAV) vector, Dr. Breakefield has been able to normalize brain structures and extend the lifespan to almost normal length in mouse models of both TSC1 and TSC2. This research was funded in part by grants through the Tuberous Sclerosis Complex Research Program (TSCRP), demonstrating how funds appropriated because of the tireless advocacy during the March on Capitol Hill has meaningfully advanced TSC research. They discuss how AAV vector technology is already being used in other diseases, what are the potential benefits of gene therapy in TSC over current treatment options, and how to transition from ongoing preclinical trials to future clinical trials.

Resources and Links
Read more about Dr. Breakefield’s Research: https://cdmrp.army.mil/tscrp/research_highlights/19breakefield_highlight
Register now for the final Virtual TSC & LAM Conference and view tracks from the previous two: https://www.tsalliance.org/news/join-us-for-the-2020-virtual-tsc-lam-conference-series/
Visit the Virtual Exhibit Hall: https://www.tsalliance.org/virtual-exhibit-hall/
Learn more about a clinical study for individuals with TSC who contracted COVID-19: https://www.tsalliance.org/tsc-matters/have-you-or-your-loved-one-with-tsc-been-diagnosed-with-covid-19-2/


Episode 3: The 2019 International TSC Research Conference



In the third episode of TSC Now, host Dan Klein, Director of Digital Platforms for the Tuberous Sclerosis Alliance, talks to the two co-chairs of the 2019 International TSC Research Conference: Changing the Course of TSC. Continue reading Episode 3: The 2019 International TSC Research Conference


Episode 1: TSC Global Day, 45th Anniversary and the Future of TSC Research



Welcome to the debut episode of TSC Now, a podcast from the Tuberous Sclerosis Alliance! In this episode Dan Klein, Director of Digital Platforms, talked to Jennifer Flinn from TS Canada ST, Eva Schoeters from Be-TSC in Belgium, and Rahul Vipparthi from the TS Alliance of India about the challenges they face in their countries, TSC Global Awareness Day and how they are spreading awareness of TSC. He also talked to Kari Luther Rosbeck, President and CEO of the Tuberous Sclerosis Alliance and Chief Scientific Officer Steve Roberds, PhD, about the organization’s 45th anniversary and history, how Unlock the Cure changed the role of the TS Alliance in driving research and what the future holds for TSC research. Finally, Dan talked to Jill Woodworth, host of TSC Talks, about why she started a podcast, what her future plans are for TSC Talks and what she’s learned through the conversations she has had with her guests.

TS Canada ST: https://www.tscanada.ca/

Be-TSC: http://www.betsc.be/

TS Alliance of India: http://tsa-india.org/

To learn more about the Educational Meeting and International TSC Research Conference in Toronto visit: https://www.tsalliance.org/news/the-2019-international-tsc-research-conference-is-june-20-22-in-toronto/

To read about the TANDem Project visit: https://www.tsalliance.org/international-tand-research-project-awarded-funding/

To read the Executive Summary of the Research Business Plan visit: https://www.tsalliance.org/about-us/

To listen to TSC Talks visit: https://www.spreaker.com/show/jill-mccutchan-woodworths-tracks

Send your questions for the Question and Answer episode to tscnow@tsalliance.org.