M. Blackwood

AAV gene therapy for Tay-Sachs disease

Douglas R Martin, T. Flotte, F. Eichler, 2022, Nature Medicine.

SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.

Derek H. Oakley, Robert H. Brown, M. Frosch, 2020, The New England journal of medicine.

CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro

Tomás C. Rodríguez, L. Petrucelli, J. Kocher, 2022, bioRxiv.

Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans

T. Flotte, C. Mueller, G. Gernoux, 2020, Molecular therapy : the journal of the American Society of Gene Therapy.

Unexpected Death of a Duchenne Muscular Dystrophy Patient in an N-of-1 Trial of rAAV9-delivered CRISPR-transactivator

B. Wong, H. Lidov, S. Pajusalu, 2023, medRxiv.

Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice

T. Flotte, C. Mueller, F. Borel, 2022, Molecular therapy. Methods & clinical development.

Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells

T. Flotte, Katelyn E. Sylvia, G. Gao, 2021, Molecular Therapy. Methods & Clinical Development.

Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques

Robert H. Brown, C. Mueller, F. Borel, 2018, Science Translational Medicine.

First-in-human AAV Gene Therapy for Tay-Sachs Disease

Douglas R Martin, T. Flotte, F. Eichler, 2021 .