Developing a High Throughput Screening Platform for Gene Therapy and Drug Discovery in ASD

There has been a strong push to test new gene therapies in autism, including use of new antisense oligonnucleotide therapy, which targets working copies of genes to increase production of its associated protein. This highly innovative approach could remove a major bottleneck in the development of gene therapies for autism by developing a new way to test genetic therapy targets genome-wide.


Stephen Tran

Jonathan Sebat