Yuhui Hu

AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease

S. Gray, Joseph R. Mazzulli, Frances C. Shaffo, 2022, The Journal of clinical investigation.

Adeno-associated viral vector serotype 9-based gene replacement therapy for SURF1-related Leigh syndrome

S. Gray, MinJae Lee, M. Rioux, 2021, Molecular therapy. Methods & clinical development.

Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation

R. Tikkanen, S. Gray, S. Cathey, 2020, Molecular therapy : the journal of the American Society of Gene Therapy.

Sustained AAV9-mediated expression of a non-self protein in the CNS of non-human primates after immunomodulation

D. Fuller, S. Gray, A. Messer, 2018, PloS one.

Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies

J. Bonifacino, M. Sahin, S. Gray, 2023, The Journal of clinical investigation.

Long-term progression of retinal degeneration in a preclinical model of CLN7 Batten disease as a baseline for testing clinical therapeutics

Katherine J. Wert, S. Gray, Emily R. Nettesheim, 2022, EBioMedicine.

Preclinical results in rodents strongly support clinical evaluation of scAAV9/ as a potential gene therapy for CLN7 patients

S. Gray, Frances C. Shaffo, J. Mazzulli, 2021 .

A Single Residue Mutation in the Gαq Subunit of the G Protein Complex Causes Blindness in Drosophila

R. Hardie, Y. Rong, Fengping Xiao, 2017, G3: Genes, Genomes, Genetics.

SMRT Sequencing Enables High-Throughput Identification of Novel AAVs from Capsid Shuffling and Directed Evolution

S. Braun, S. Gray, M. Kanchwala, 2023, Genes.