Heather Gray‐Edwards

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

Julia F. Alterman, Bruno M.D.C. Godinho, Matthew Hassler et al. · Nature Biotechnology · 2019 · 220 citations

Molecular Neuroscience, Signal Transduction, Medicine +10

Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide

Hélène Tran, Michael P. Moazami, Huiya Yang et al. · Nature Medicine · 2021 · 166 citations · Full text

Protein Expression, Biochemistry, Natural Sciences +6

AAV gene therapy for Tay-Sachs disease

Terence R. Flotte, Oğuz Çataltepe, Ajit S Puri et al. · Nature Medicine · 2022 · 122 citations · Full text

Gene Therapies, Aav Gene Therapy, Pathogenesis +5

Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector

Sourav Choudhury, Anne F Harris, Damien J. Cabral et al. · Molecular Therapy · 2015 · 113 citations · Full text

In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy

Sourav Choudhury, Zachary Fitzpatrick, Anne F Harris et al. · Molecular Therapy · 2016 · 113 citations · Full text