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Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models

[DOI] [PMC free article] [PubMed] [Google Scholar] 210.Hedesan OC, Fenzl A, Digruber A, Spirk K, Baumgartner-Parzer S, Bilban M, Kenner L, Vierhapper M, Elbe-Brger A, Kiefer FW

Consequently, the main factors affecting the production of L-carnitine with both wild type and genetically engineered E
