Nettie K. Pyne

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

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 · 69 citations · Full text

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 · 65 citations · Full text

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 · 31 citations · Full text

Aberrant Splicing in Transgenes Containing Introns, Exons, and V5 Epitopes: Lessons from Developing an FSHD Mouse Model Expressing a D4Z4 Repeat with Flanking Genomic Sequences

Eugénie Ansseau, Jacqueline S. Domire, Lindsay M. Wallace et al. · PLoS ONE · 2015 · 16 citations · Full text

Aberrant Splicing, Genetics, Genomic Mechanism +18