Article

Programmable Repair of Disease-Causing UGA Stop Codons in Mammalian Brain

2026-05-16

Abstract excerpt

<h4>ABSTRACT</h4> Protein truncating variants caused by UGA stop codons are the most prevalent class of rare variant mutations in neurodevelopmental diseases. Suppressor transfer RNA (sup-tRNA) have therapeutic potential for premature termination codon (PTC) repair, but have thus far underperformed by traditional AAV delivery platforms and progress has been hampered by the lack of methods to non-invasively assess...

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Literature Corpus work: 251a8a35-0255-5019-97a1-f2618786c6a7
DOI: 10.64898/2026.05.13.724978

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