Suchergebnisse - "Flanigan, Kevin M"

PubPharm (275)

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

Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease

enthalten in: Molecular therapy : the journal of the American Society of Gene Therapy | 2024

von Herstine, J. | Chang, P. | Chornyy, S. | +14

3

Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications : An Open-label Study

enthalten in: Journal of neuromuscular diseases | 2024

von Nicolau, S. | Malhotra, J. | Kaler, M. | +10

4

Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model

enthalten in: Molecular therapy. Methods & clinical development | 2023

von Gushchina, L. | Bradley, A. | Vetter, T. | +5

5

Exome sequencing in the pediatric neuromuscular clinic leads to more frequent diagnosis of both neuromuscular and neurodevelopmental conditions

enthalten in: Muscle & nerve | 2023

von Meyer, A. | Ma, J. | Brock, G. | +9

6

CRISPR-Cas9 homology-independent targeted integration of exons 1-19 restores full-length dystrophin in mice

enthalten in: Molecular therapy. Methods & clinical development | 2023

von Stephenson, A. | Nicolau, S. | Vetter, T. | +7

7

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

8

Inherited myopathy plus : Double-trouble from rare neuromuscular disorders

enthalten in: Neuromuscular disorders : NMD | 2023

von Granger, A. | Beecher, G. | Liewluck, T. | +4

9

Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines

enthalten in: Frontiers in cell and developmental biology | 2023

von Almeida, C. | Robriquet, F. | Vetter, T. | +9

10

Exome sequencing in the pediatric neuromuscular clinic leads to more frequent diagnosis of both neuromuscular and neurodevelopmental conditions

enthalten in: Muscle & Nerve | 2023

von Meyer, A. | Ma, J. | Brock, G. | +9

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