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 · 216 citations · Full text

Medicine, Ophthalmology, Vision Science +15

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 · 166 citations · Full text

Life Sciences, Genetics, Molecular Biology +11

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 · 56 citations

Life Sciences, Genetics, 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 · 26 citations · Full text

Life Sciences, Cell Biology, Molecular Genetics +13

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 · 14 citations

Life Sciences, Genetics, Molecular Biology +17