Regulating PCCA gene expression by modulation of pseudoexon splicing patterns to rescue enzyme activity in propionic acidemia
Open Access
- 13 December 2023
- journal article
- research article
- Published by Elsevier in Molecular Therapy Nucleic Acids
- Vol. 35 (1) , 102101
Abstract
No abstract availableKeywords
Funding Information
- Ministerio de Ciencia e Innovación
- European Regional Development Fund
- Fundación Ramón Areces
- Novo Nordisk Fonden (PID2019-105344RB-I00/AEI/10.13039/501100011033)
- Sundhed og Sygdom, Det Frie Forskningsråd (9039-00281B)
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