Gene Replacement Therapy Update

Drs. Henry H. C. Lee, Itay Tokatly Latzer, Mariarita Bertoldi, Guangping Gao, Phillip L. Pearl, Mustafa Sahin, and Alexander Rotenberg teamed up to create a paper on the status of their Gene Replacement Therapy project results using the SSADH mouse model.  This article title is Gene replacement therapies for inherited disorders of neurotransmission: Current progress in succinic semialdehyde dehydrogenase deficiency. The article has been published in the Journal of Inherited Metabolic Disease (JIMD).

The staff at JIMD are working with Dr. Lee and Rotenberg to create a visual abstract, Podcast, and a YouTube presentation about this very important work.

We remain grateful to the team of doctors working on finding a treatment for those struggling with SSADH Deficiency. 

Gene Replacement Therapies For Inherited Disorders