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Allele-specific silencing as treatment for gene duplication disorders: proof-of-principle in autosomal dominant leukodystrophy.
Brain. 2019 Jul 1;142(7):1905-1920. doi: 10.1093/brain/awz139.
Brain. 2019.
PMID: 31143934
Free article.
A high-content drug screening strategy to identify protein level modulators for genetic diseases: A proof-of-principle in autosomal dominant leukodystrophy.
Giorgio E, Pesce E, Pozzi E, Sondo E, Ferrero M, Morerio C, Borrelli G, Della Sala E, Lorenzati M, Cortelli P, Buffo A, Pedemonte N, Brusco A.
Giorgio E, et al. Among authors: della sala e.
Hum Mutat. 2021 Jan;42(1):102-116. doi: 10.1002/humu.24147. Epub 2020 Dec 8.
Hum Mutat. 2021.
PMID: 33252173
Free article.
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