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, Post Office Box 348626, Sacramento, CA 95834
New Research on Gene Therapy and Lafora Body Aggregation in Mice
/by Christine KellyDr. Berge Minassian’s lab has developed and tested an AAV gene therapy for Lafora Disease to reduce glycogen synthesis and halt Lafora Body aggregation in mice.
We are still a long way from being able to use gene therapy to treat Lafora Disease, however, this is an important step toward curing it for future generations.
If you have questions about gene therapy and Lafora Disease, check out our easy-to-read guide linked below. Please feel free to contact our Science Director, Dr. Kit Donohue, at katherine@chelseashope.org.
New Research on Gene Therapy and Lafora Disease
/by Christine KellyResearchers 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
/by Christine KellyQuestions 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.
Patient Community Meet & Greet with New Science Director
/by Christine KellyPlease join our new Science Director, Dr. Kit Donohue, for a community meet-and-greet. As Kit steps into her new role, she would like to hear from you—the patient community!
Kit has been fortunate to study in Dr. Matthew Gentry’s lab for the past five years, where she met and collaborated with many of the scientists seeking treatment for Lafora Disease. The progress that has already been made is a testament to what can happen when scientists work closely with the patient community.
Kit is excited to contribute to deepening that collaboration as we move closer to finding a cure for Lafora Disease. We hope you will join us and share your needs, questions, concerns, and hopes for the future of our community.
When: June 29, 2022 06:00 PM in Eastern Time US and Canada. Registration before June 29, 2022 is required.
If you are unable to attend or would like to submit a question/thought ahead of time or privately, please email katherine@chelseashope.org.