Patricia Biasutto

Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect

Artur V. Cideciyan, Samuel G. Jacobson, Arlene V. Drack et al. · Nature Medicine · 2018 · 209 citations · Full text

Leber Congenital Amaurosis, Photoreceptor Cilium Defect, Ocular Disease +10

Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models

Kalyan Dulla, Mònica Aguilà, Amelia Lane et al. · Molecular Therapy — Nucleic Acids · 2018 · 162 citations · Full text

G Lca10 Models, Transcriptional Regulation, Natural Sciences +8

Primary Microcephaly, Impaired DNA Replication, and Genomic Instability Caused by Compound Heterozygous<i>ATR</i>Mutations

Houda Benhelli‐Mokrani, Laetitia Gaillard, Patricia Biasutto et al. · Human Mutation · 2012 · 55 citations

Genetics, Genomic Mechanism, Molecular Genetics +17

Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis

Wouter Beumer, Jim Swildens, Teresinha Leal et al. · PLoS ONE · 2019 · 24 citations · Full text

Inflammatory Lung Disease, Molecular Biology, Cellular Physiology +17

WS01.2 QR-010, an RNA therapy, restores CFTR function using in vitro and in vivo models of ΔF508 CFTR

Wouter Beumer, Jim Swildens, Noreen Henig et al. · Journal of Cystic Fibrosis · 2015 · 12 citations

Rna Therapy, Molecular Physiology, Natural Sciences +9