Three-Dimensional Model for Identifying Fragile X Treatments

Three-Dimensional Model for Identifying Fragile X Treatments
With a $90,000 grant from FRAXA Research Foundation awarded in 2018, Dr. Peng Jin and Dr. Juhnee Kang at Emory University will develop and analyze fragile X brain organoids to understand the disorder and identify treatment targets. $90,000 Grant Peng Jin, PhD Principal Investigator Yunhee Kang, PhD FRAXA Postdoctoral Fellow Zhexing Wen, PhD Co-Principal Investigator Emory University School of Medicine 2018 FRAXA Research Grant $90,000 over 2 Years by Peng Jin One of the most exciting recent advancements in stem cell research has been the development of a new model that bridges the gap between mice and humans. Chosen as 2017 Method of the Year by Nature, organoids are tiny, self-organized, three-dimensional tissue cultures grown from stem cells. They can be crafted to model much of the complexity of any organ — even the brain. This new technique affords the opportunity to evaluate potential drug treatments in a model that more closely mimics the humanRead more

Small Molecule Modulators of Lithium for Treatment of Fragile X Syndrome

Small Molecule Modulators of Lithium for Treatment of Fragile X Syndrome

With a $219,500 grant from FRAXA Research Foundation, Dr. Stephen Haggarty from Havard/MIT developed a high-throughput drug screen to find compounds that inhibit GSK3, a critical enzyme in fragile X. He looked for compounds that can accomplish this either alone or in combination with lithium, offering the possibility of enhancing the effectiveness of lithium as a treatment. His drug screen used patient-specific neural progenitor (NP) cells derived from human induced pluripotent stem cells (iPSCs) – which are created from cells in a skin biopsy from people with fragile X syndrome (FXS) and other autism spectrum disorders.

Read more