Modulating splicing in 5’ untranslated regions to treat rare haploinsufficient disease

Beer Wells ES., De Conti L., Kim HC., Rohani N., Chundru K., Watts LM., Dawes R., Chen Y., Martin-Geary AC., Griffiths MJ., Scott S., Bamford RA., Mill J., Wright CF., Baralle M., Sanders SJ., Whiffin N.

DOI

10.64898/2025.12.07.692584

Type

Preprint

Publication Date

2025-12-09T00:00:00+00:00

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