Clubhouse Podcast
Listen to Alok Tayi and Joshua Forman discuss their company Vibe Bio and their fight against rare disease on the Gene Fixers podcast. We are glad to partner with Vibe Bio!
New Research on Gene Therapy and Lafora Disease
Researchers present gene replacement therapy as an approach for Lafora disease in the recently published article “Malin restoration as proof of concept for gene therapy for Lafora disease.”
This paper shows that restoring malin expression in a mouse model that lacks it can reduce Lafora Body accumulation and neuroinflammation.
This proof-of-concept suggests that it would be beneficial to develop gene therapy for restoring malin expression in patients where malin is mutated or missing.
If you have questions about gene therapy and Lafora Disease, please feel free to contact our Science Director, Dr. Kit Donohue, at katherine@chelseashope.org.
Gene Therapy 101
Questions about gene therapy and Lafora Disease? Access the easy-to-read guide Gene Therapy 101 today.
If you want to read more about gene therapy, this is a good patient-friendly website.
If you have more questions about gene therapy and Lafora Disease, please feel free to contact our Science Director, Dr. Kit Donohue, at katherine@chelseashope.org.
Access the PDF in Italian, too: L’ABC della terapia genica.
The mission of Chelsea’s Hope is to improve the lives of those affected by Lafora Disease and help accelerate the development of treatments.
Chelsea’s Hope Lafora Children Research Fund is an IRS 501(c)3 non-profit organization. EIN: 27-1008382
Chelsea’s Hope c/o Dr. Donohue
976 Maywick Dr.
Lexington, KY 40504
