All About Gene Therapy

In this session, we will learn what gene therapy is, the different types of gene therapy, and how gene therapy could be used to treat Ataxia in the future. We will also discuss the limitations of gene therapy technology, as well as what research is being done right now on gene therapy for Ataxia. If you have questions about gene therapy, this webinar is for you.
For more information on Ataxia, please visit our website: www.ataxia.org
Become a Free NAF member! bit.ly/JoinNAF
Follow us on Social Media!
NAF Facebook: / ataxiafounda. .
NAF Twitter: / naf_ataxia
NAF Instagram: / ataxiafound. .
Ataxia Subreddit: / ataxia
About the Speaker-
Speaker: Carolyn Yrigollen, PhD
Institution: Children's Hospital of Philadelphia
Dr. Yrigollen is a researcher at the Children’s Hospital of Philadelphia with 10 years of experience working on gene editing systems to treat heritable neurological disorders. Carolyn earned her Ph.D. in genetics from the University of California, Davis where she studied the structure and function of the Fragile X gene. She is currently working on CRISPR based approaches to correct the underlying mutations leading to Fragile X Syndrome and Fragile X-associated Tremor/Ataxia Syndrome.

[@bricoleuse]

Thank you for this comprehensive discussion about gene therapy and ataxia. These are exciting times we live in! I am personally curious to hear more about ASO SCA2 trials involving BIIB105 in the future :) On behalf of everyone affected by the ataxias, thank you for all your efforts on our behalf!

[@cky5573]

Thank you for your research. I know research takes many years before any kind of treatment is available to the public. I think any kind of treatment will be too late for my family member, but hopefully others will benefit.

[@FredFlinstone-ic5op]

i have olivopontocerebellar sca34