Search Page
Save citations to file
Email citations
Send citations to clipboard
Add to Collections
Add to My Bibliography
Create a file for external citation management software
Your saved search
Your RSS Feed
Search Results
7 results
Filters applied: . Clear all
Results are displayed in a computed author sort order.
The Results By Year timeline is not available.
Page 1
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.
PLoS One. 2015 Mar 5;10(3):e0118813. doi: 10.1371/journal.pone.0118813. eCollection 2015.
PLoS One. 2015.
PMID: 25742305
Free PMC article.
Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD.
Wallace LM, Saad NY, Pyne NK, Fowler AM, Eidahl JO, Domire JS, Griffin DA, Herman AC, Sahenk Z, Rodino-Klapac LR, Harper SQ.
Wallace LM, et al. Among authors: pyne nk.
Mol Ther Methods Clin Dev. 2017 Dec 24;8:121-130. doi: 10.1016/j.omtm.2017.12.005. eCollection 2018 Mar 16.
Mol Ther Methods Clin Dev. 2017.
PMID: 29387734
Free PMC article.
Item in Clipboard
AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD.
Giesige CR, Wallace LM, Heller KN, Eidahl JO, Saad NY, Fowler AM, Pyne NK, Al-Kharsan M, Rashnonejad A, Chermahini GA, Domire JS, Mukweyi D, Garwick-Coppens SE, Guckes SM, McLaughlin KJ, Meyer K, Rodino-Klapac LR, Harper SQ.
Giesige CR, et al. Among authors: pyne nk.
JCI Insight. 2018 Nov 15;3(22):e123538. doi: 10.1172/jci.insight.123538.
JCI Insight. 2018.
PMID: 30429376
Free PMC article.
Item in Clipboard
Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models.
Morelli KH, Griffin LB, Pyne NK, Wallace LM, Fowler AM, Oprescu SN, Takase R, Wei N, Meyer-Schuman R, Mellacheruvu D, Kitzman JO, Kocen SG, Hines TJ, Spaulding EL, Lupski JR, Nesvizhskii A, Mancias P, Butler IJ, Yang XL, Hou YM, Antonellis A, Harper SQ, Burgess RW.
Morelli KH, et al. Among authors: pyne nk.
J Clin Invest. 2019 Dec 2;129(12):5568-5583. doi: 10.1172/JCI130600.
J Clin Invest. 2019.
PMID: 31557132
Free PMC article.
Item in Clipboard
RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model.
Aimiuwu OV, Fowler AM, Sah M, Teoh JJ, Kanber A, Pyne NK, Petri S, Rosenthal-Weiss C, Yang M, Harper SQ, Frankel WN.
Aimiuwu OV, et al. Among authors: pyne nk.
Mol Ther. 2020 Jul 8;28(7):1706-1716. doi: 10.1016/j.ymthe.2020.04.007. Epub 2020 Apr 16.
Mol Ther. 2020.
PMID: 32353324
Free PMC article.
Item in Clipboard
Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease.
Herstine JA, Chang PK, Chornyy S, Stevenson TJ, Sunshine AC, Nokhrina K, Rediger J, Wentz J, Vetter TA, Scholl E, Holaway C, Pyne NK, Bratasz A, Yeoh S, Flanigan KM, Bonkowsky JL, Bradbury AM.
Herstine JA, et al. Among authors: pyne nk.
Mol Ther. 2024 Mar 27:S1525-0016(24)00212-0. doi: 10.1016/j.ymthe.2024.03.034. Online ahead of print.
Mol Ther. 2024.
PMID: 38549375
Item in Clipboard
Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy.
Bradbury AM, Bagel J, Swain G, Miyadera K, Pesayco JP, Assenmacher CA, Brisson B, Hendricks I, Wang XH, Herbst Z, Pyne N, Odonnell P, Shelton GD, Gelb M, Hackett N, Szabolcs P, Vite CH, Escolar M.
Bradbury AM, et al.
Mol Ther. 2024 Jan 3;32(1):44-58. doi: 10.1016/j.ymthe.2023.11.014. Epub 2023 Nov 11.
Mol Ther. 2024.
PMID: 37952085
Item in Clipboard
Cite
Cite