FRAXA’s primary mission is to speed up progress towards effective treatments and a cure for Fragile X. As “bottlenecks” are identified we will try to facilitate solutions and add resources for university and pharma researchers worldwide, so please contact us for additional scientific resource requests.
Models and Reagents
This model contains a deletion of the Fragile X mental retardation 1 gene (Fmr1). Mutations in Fmr1 result in Fragile X syndrome, the leading monogenic cause of autism, making this rat useful for the study of both Fragile X syndrome and autism.
The National Human Neural Stem Cell Resource (SCR) provides to the research community neural stem cells harvested from the post-natal, post-mortem, human brain. Dr. Philip Schwartz directs this resource at the Children’s Hospital of Orange County (CHOC) Research Institute.
Human cortical neural stem cells that carry the Fragile X mutation are available for distribution to interested researchers. These cells are grown as neurospheres and are mainly neural progenitor cells. They can be differentiated into neurons and astrocytes that lack FMRP with long-term culturing. For more information about these cells please refer to Svendsen et al., J Neuroscience Methods 85:141-163 (1998), and contact Dr. Anita Bhattacharyya at the University of Wisconsin-Madison Waisman Center.