E. Frair
Nationwide Children's Hospital(US)
Publications by Year
Research Areas
Muscle Physiology and Disorders, RNA Research and Splicing, RNA Interference and Gene Delivery, Virus-based gene therapy research, CRISPR and Genetic Engineering
Most-Cited Works
- → PRMT5–PTEN molecular pathway regulates senescence and self-renewal of primary glioblastoma neurosphere cells(2016)127 cited
- → Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping(2021)39 cited
- → A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2(2022)31 cited
- → Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse(2022)26 cited
- → Automated immunofluorescence analysis for sensitive and precise dystrophin quantification in muscle biopsies(2021)21 cited
- → CRISPR-Cas9 homology-independent targeted integration of exons 1–19 restores full-length dystrophin in mice(2023)17 cited
- → Absence of Significant Off-Target Splicing Variation with a U7snRNA Vector Targeting DMD Exon 2 Duplications(2021)15 cited
- → Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model(2023)14 cited
- → Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy(2022)13 cited
- → Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study(2024)7 cited