Antisense Oligonucleotide-Based Rescue of Complex Intronic Splicing Defects in <i>ABCA4</i>-Reference-Cited by-同舟云学术

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Antisense Oligonucleotide-Based Rescue of Complex Intronic Splicing Defects in ABCA4

Published:2024-06-01 Issue:3 Volume:34 Page:125-133
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Corradi Zelia¹,Hitti-Malin Rebekkah J.¹,de Rooij Laura A.¹,Garanto Alejandro¹²^ORCID,Collin Rob W. J.¹,Cremers Frans P. M.¹

Affiliation:

1. Department of Human Genetics, Radboud University Medical Center, Nijmegen, The Netherlands.

2. Department of Pediatrics, Amalia Children’s Hospital, Radboud University Medical Center, Nijmegen, The Netherlands.

Publisher

Mary Ann Liebert Inc

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2024.0008

Reference40 articles.

1. ABCA4-Associated Retinal Degenerations Spare Structure and Function of the Human Parapapillary Retina

2. A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Starqardt macular dystrophy

3. ABCR expression in foveal cone photoreceptors and its role in Stargardt macular dystrophy

4. The 220-kDa Rim Protein of Retinal Rod Outer Segments Is a Member of the ABC Transporter Superfamily

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