SLC1A4 Deficiency is a devastating neurological disorder affecting Jewish families around the world. Roughly 1 out of 100 Ashkenazi Jews are carriers and most don't know it. Together, we can eradicate this genetic condition.
Given its genetic characteristics, researchers believe that SLC1A4 Deficiency is a promising candidate for gene therapy. SLC1A4 Deficiency should also be added to Ashkenazi Jewish genetic testing panels to reduce its incidence, just as other Jewish genetic conditions such as Tay-Sachs Disease have been drastically reduced as a result of increased carrier screening.
This first funding milestone starts work on creating the tools needed to test future therapies.
The program will progress through the gene therapy development, animal studies, toxicology, and human trials.
Donations to the SLC1A4 Initiative are collected through Boston Children's Hospital, a 501(c)(3) organization, and are tax-deductible in the United States to the extent permitted by law.