Understanding and Rescuing the Splicing Defect Caused by the Frequent ABCA4 Variant c.4253+43G>A Underlying Stargardt Disease

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Authors: Nuria Suárez-Herrera, Alejandro Garanto, Rob W J Collin

Journal title: Nucleic Acid Therapeutics

Journal number: vol. 34, No. 2

Journal publisher: Mary Ann Liebert Inc.

Published year: 2024

DOI identifier: 10.1089/nat.2023.0076

ISSN: 2159-3337