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Preclinical Development of Antisense Oligonucleotides to Rescue Aberrant Splicing Caused by an Ultrarare <i>ABCA4</i> Variant in a Child with Early-Onset Stargardt Disease

Precision medicine is rapidly gaining recognition in the field of (ultra)rare conditions, where only a few individuals in the world are affected. Clinical trial design for a small number of patients is extremely challenging, and for this reason, the development of N-of-1 strategies is explored to ac...

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Principais autores: Nuria Suárez-Herrera, Catherina H. Z. Li, Nico Leijsten, Dyah W. Karjosukarso, Zelia Corradi, Femke Bukkems, Lonneke Duijkers, Frans P. M. Cremers, Carel B. Hoyng, Alejandro Garanto, Rob W. J. Collin
Format: Artigo
Sprog:Inglês
Udgivet: MDPI AG 2024-03-01
Serier:Cells
Fag:
N-of-1
antisense oligonucleotide
splicing modulation
pseudoexon
RNA therapy
<i>ABCA4</i>
Online adgang:https://www.mdpi.com/2073-4409/13/7/601
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