Following the publication of his team’s gene therapy work with AAV-9 in spinal muscular atrophy (SMA) mouse models (Foust et al., Nature Biotechnology), Dr. Brian Kaspar participated in Nationwide Children’s Hospital’s monthly podcast about neuromuscular research. In this podcast, Dr. Kaspar spoke about a number of topics including:
- What is spinal muscular atrophy (SMA)
- The genetics of SMA
- The difficulty developing treatments for SMA
- The results AAV-9 has shown in SMA mouse models
- The need for additional studies before moving to human trials
- The next steps toward the clinic
The podcast is available by clicking here and scrolling to the May podcast. At the time of this post, there is a problem in how the podcast is available on the Nationwide Children’s Hospital website. The audio file has a comma added at the end of the file type which prevents players from recognizing file type. In order to play the podcast, simply download it to your computer, select the file name, and remove the comma. Your computer should then recognize the podcast as a MP3 file.
Recently, FightSMA launched the “Realizing the Dream” fundraising campaign which partners with the Gwendolyn Strong Foundation to advance a common goal: to bring SMA gene therapy to clinical trial. These efforts will support Dr. Kaspar and other collaborating scientists. To learn more about the gene therapy campaign and how you can get involved, click here.
