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ArticlesPeer ReviewedFull Text
Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect
Author / Creator:Cideciyan, Artur V. ; Jacobson, Samuel G. ; Drack, Arlene V. ; Ho, Allen C. ; Charng, Jason ; Garafalo, Alexandra V. ; Roman, Alejandro J. ; Sumaroka, Alexander ; Han, Ian C. ; Hochstedler, Maria D. ; Pfeifer, Wanda L. ; Sohn, Elliott H. ; Taiel, Magali ; Schwartz, Michael R. ; Biasutto, Patricia ; Wit, Wilma de ; Cheetham, Michael E. ; Adamson, Peter ; Rodman, David M. ; Platenburg, Gerard ; Tome, Maria D. ; Balikova, Irina ; Nerinckx, Fanny ; Zaeytijd, Julie De ; Van Cauwenbergh, Caroline ; Leroy, Bart P. ; Russell, Stephen R.
Publisher:New York: Nature Publishing Group US
Journal title:Nature medicine, 2019-02, Vol.25 (2), p.225-228
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Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis
Author / Creator:Beumer, Wouter ; Swildens, Jim ; Leal, Teresinha ; Noel, Sabrina ; Anthonijsz, Herma ; van der Horst, Geert ; Kuiperij-Boersma, Hester ; Potman, Marko ; van Putten, Charlotte ; Biasutto, Patricia ; Platenburg, Gerard ; de Jonge, Hugo ; Henig, Noreen ; Ritsema, Tita
Publisher:United States: Public Library of Science
Journal title:PloS one, 2019-06, Vol.14 (6), p.e0219182
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Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models
Author / Creator:Dulla, Kalyan ; Aguila, Monica ; Lane, Amelia ; Jovanovic, Katarina ; Parfitt, David A. ; Schulkens, Iris ; Chan, Hee Lam ; Schmidt, Iris ; Beumer, Wouter ; Vorthoren, Lars ; Collin, Rob W.J. ; Garanto, Alejandro ; Duijkers, Lonneke ; Brugulat-Panes, Anna ; Semo, Ma’ayan ; Vugler, Anthony A. ; Biasutto, Patricia ; Adamson, Peter ; Cheetham, Michael E.
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy. Nucleic acids, 2018-09, Vol.12, p.730-740
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Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.29...
Author / Creator:Kalyan Dulla ; Monica Aguila ; Amelia Lane ; Katarina Jovanovic ; David A. Parfitt ; Iris Schulkens ; Hee Lam Chan ; Iris Schmidt ; Wouter Beumer ; Lars Vorthoren ; Rob W.J. Collin ; Alejandro Garanto ; Lonneke Duijkers ; Anna Brugulat-Panes ; Ma’ayan Semo ; Anthony A. Vugler ; Patricia Biasutto ; Peter Adamson ; Michael E. Cheetham
Publisher:Elsevier
Journal title:Molecular therapy. Nucleic acids, 2018-09, Vol.12, p.730-740
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Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect

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

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

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Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.29...

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