Concepedia
Statistics
Publications
3
Citations
110
H-Index
Active since
2019
Affiliations
University College London(Current)
NIHR Great Ormond Street Hospital Biomedical Research Centre
Great Ormond Street Hospital
National Institute of Neurological Disorders and Stroke
Sara Aguti is an author at University College London.
Publications per year
2019–2024
A recurrent COL6A1 pseudoexon insertion causes muscular dystrophy and is effectively targeted by splice-correction therapies
Véronique Bolduc, A. Reghan Foley, Herimela Solomon-Degefa et al. · JCI Insight · 2019 · 64 citations · Full text
Exon-Skipping Oligonucleotides Restore Functional Collagen VI by Correcting a Common COL6A1 Mutation in Ullrich CMD
Sara Aguti, Véronique Bolduc, Pierpaolo Ala et al. · Molecular Therapy — Nucleic Acids · 2020 · 34 citations · Full text
Strategies to improve the design of gapmer antisense oligonucleotide on allele-specific silencing
Sara Aguti, Shuzhi Cheng, Pierpaolo Ala et al. · Molecular Therapy — Nucleic Acids · 2024 · 12 citations · Full text
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