A different type of genetic therapy: correcting a defective gene using antisense oligonucleotide treatment in CEP290 p.Cys998X LCA

Arlene V Drack; Stephen R Russell; Artur V Cideciyan; Samuel G Jacobson; Bart P Leroy; Wanda L Pfeifer; Alina V Dumitrescu; Alexandra V Garafolo; Allen C Ho; Caroline Van Cauwenbergh; Julie De Zaeytijd; Wil den Hollander; Michael R Schwartz; Fredrich Asmus; Aniz Girach

doi:10.1016/j.jaapos.2021.08.021

Abstract

A different type of genetic therapy: correcting a defective gene using antisense oligonucleotide treatment in CEP290 p.Cys998X LCA

Arlene V Drack, Stephen R Russell, Artur V Cideciyan, Samuel G Jacobson, Bart P Leroy, Wanda L Pfeifer, Alina V Dumitrescu, Alexandra V Garafolo, Allen C Ho, Caroline Van Cauwenbergh, …

Journal of AAPOS, Vol.25(4), p.e5

08/2021

DOI: 10.1016/j.jaapos.2021.08.021

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Details

Title: Subtitle: A different type of genetic therapy: correcting a defective gene using antisense oligonucleotide treatment in CEP290 p.Cys998X LCA
Creators: Arlene V Drack
Stephen R Russell
Artur V Cideciyan
Samuel G Jacobson
Bart P Leroy
Wanda L Pfeifer
Alina V Dumitrescu
Alexandra V Garafolo
Allen C Ho
Caroline Van Cauwenbergh
Julie De Zaeytijd
Wil den Hollander
Michael R Schwartz
Fredrich Asmus
Aniz Girach
Resource Type: Abstract
Publication Details: Journal of AAPOS, Vol.25(4), p.e5
DOI: 10.1016/j.jaapos.2021.08.021
ISSN: 1091-8531
eISSN: 1528-3933
Language: English
Date published: 08/2021
Academic Unit: Stead Family Department of Pediatrics; Ophthalmology and Visual Sciences
Record Identifier: 9984187176102771

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