Family Research Foundations: OUR STRATEGIC PARTNERS
GEN, London, works close partnership with a number of Family Research Organizations, to take the results of our research rapidly into the clinic, with the aim of developing better treatments for rare disease patients.
Pitt Hopkins Research Foundation (PTHS) Projects
in vivo gene therapy
In collaboration with Alysson Muotri’s lab
Institute for Genomic Medicine, Rady Children’s Hospital, Sanford Consortium, Ca, USA
Project objective: To elucidate the effects of the lack of the TCF4 gene and its re-introduction on the embryonic and postnatal development of the brain cortex.