Wilson's research involves the development of viral-based gene therapies for genetic diseases. A major research focus is the generation of novel vectors for improved transduction efficiencies and regulated expression, as well as the elucidation of host immune responses to viral vectors. His work emphasizes the creation of vectors for
in vivo gene therapy concentrating on
adeno-associated viruses. This work began with the discovery in his laboratory of a new family of primate AAVs; over 120 new AAV capsids were rescued as latent genomes from primate tissues and studied for their biology and potential as vectors. This has led to an enhanced understanding of vector host interactions and a new generation of vectors with substantially improved performance profiles beyond that provided from the original 6 AAV isolates. More recently, Wilson's laboratory has used AAV to accomplish successful
in vivo genome editing. As of 2018, his laboratory's
translational research portfolio included more than 30 orphan disease programs. The
adeno-associated viruses serotypes discovered in Wilson's lab were used in several clinically approved gene therapies including
onasemnogene abeparvovec which uses AAV9. Wilson is the founder of several gene therapy companies including Genovo, Regenxbio, Passage Bio, and Scout Bio. Gemma Biotherapeutics will aim to build advanced medicines for patients with rare diseases. Franklin Biolabs will serve as a contract research organization for companies working on genetic medicines. ==References==