Suchergebnisse - "Simmons, Tabatha R"

PubPharm (12)

1

A genome-wide association analysis of loss of ambulation in dystrophinopathy patients suggests multiple candidate modifiers of disease severity

enthalten in: bioRxiv.org | 2024

von Flanigan, K. | Waldrop, M. | Martin, P. | +9

2

A genome-wide association analysis of loss of ambulation in dystrophinopathy patients suggests multiple candidate modifiers of disease severity

enthalten in: European journal of human genetics : EJHG | 2023

von Flanigan, K. | Waldrop, M. | Martin, P. | +9

CommentIn: Eur J Hum Genet. 2023 Jun;31(6):609-610. - PMID 37291406

3

Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy

enthalten in: Molecular therapy. Nucleic acids | 2022

von Gushchina, L. | Vetter, T. | Frair, E. | +10

4

A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2

enthalten in: Molecular therapy. Methods & clinical development | 2022

von Flanigan, K. | Vetter, T. | Simmons, T. | +12

5

Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse

enthalten in: Molecular therapy. Methods & clinical development | 2022

von Wein, N. | Vetter, T. | Vulin, A. | +10

6

Candidate gene modifiers of dystrophinopathy identified by the uniform application of genome-wide datasets to novel GWAS-identified loci

enthalten in: bioRxiv.org | 2021

von Flanigan, K. | Waldrop, M. | Martin, P. | +9

7

Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping

enthalten in: Human gene therapy | 2021

von Gushchina, L. | Frair, E. | Rohan, N. | +8

8

Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping

enthalten in: Molecular therapy. Methods & clinical development | 2021

von Simmons, T. | Vetter, T. | Huang, N. | +3

9

The first exon duplication mouse model of Duchenne muscular dystrophy : A tool for therapeutic development

enthalten in: Neuromuscular disorders : NMD | 2015

von Vulin, A. | Wein, N. | Simmons, T. | +5

10

The first exon duplication mouse model of Duchenne muscular dystrophy: A tool for therapeutic development

enthalten in: Neuromuscular disorders | 2015

von Vulin, A. | Wein, N. | Simmons, T. | +5