E. Lykken

Investigating Immune Responses to the scAAV9-HEXM Gene Therapy Treatment in Tay–Sachs Disease and Sandhoff Disease Mouse Models

P. Thompson, S. Gray, V. Zaric, 2021, International journal of molecular sciences.

Recent progress and considerations for AAV gene therapies targeting the central nervous system

S. Gray, E. Lykken, Charles Shyng, 2018, Journal of Neurodevelopmental Disorders.

Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.

C. Vite, E. Bongarzone, D. Ory, 2020, The Journal of clinical investigation.