Developing antisense oligonucleotides for a TECPR2 mutation-induced, ultra-rare neurological disorder using patient-derived cellular models
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Molecular Medicine
Reference35 articles.
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1. Integrating non-mammalian model organisms in the diagnosis of rare genetic diseases in humans;Nature Reviews Genetics;2023-07-25
2. Antisense Oligonucleotide Therapy for the Nervous System: From Bench to Bedside with Emphasis on Pediatric Neurology;Pharmaceutics;2022-11-05
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