Nettie K. Pyne

Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models

Kathryn H. Morelli, Laurie B. Griffin, Nettie K. Pyne et al. · Journal of Clinical Investigation · 2019 · 75 citations · Full text

Medicine, Genetics, Neurology +17

AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD

Carlee R. Giesige, Lindsay M. Wallace, Kristin N. Heller et al. · JCI Insight · 2018 · 74 citations · Full text

Medicine, Genetics, Molecular Genetics +14

Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD

Lindsay M. Wallace, Nizar Y. Saad, Nettie K. Pyne et al. · Molecular Therapy — Methods & Clinical Development · 2017 · 68 citations · Full text

Pharmacy, Molecular Biology, Molecular Genetics +16

Human genome meeting 2016

A. K. Srivastava, Yuanyuan Wang, Raymond Y. Huang et al. · Human Genomics · 2016 · 43 citations · Full text

Life Sciences, Bioinformatics, Genetics +11

RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model

Osasumwen V. Aimiuwu, Allison M. Fowler, Megha Sah et al. · Molecular Therapy · 2020 · 36 citations · Full text

Medicine, Genetics, Neuroscience +17