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Allele-specific silencing as treatment for gene duplication disorders: proof-of-principle in autosomal dominant leukodystrophy.
Giorgio E, Lorenzati M, Rivetti di Val Cervo P, Brussino A, Cernigoj M, Della Sala E, Bartoletti Stella A, Ferrero M, Caiazzo M, Capellari S, Cortelli P, Conti L, Cattaneo E, Buffo A, Brusco A. Giorgio E, et al. Among authors: lorenzati m. 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: lorenzati m. Hum Mutat. 2021 Jan;42(1):102-116. doi: 10.1002/humu.24147. Epub 2020 Dec 8. Hum Mutat. 2021. PMID: 33252173 Free article.
Modeling primary microcephaly with human brain organoids reveals fundamental roles of CIT kinase activity.
Pallavicini G, Moccia A, Iegiani G, Parolisi R, Peirent ER, Berto GE, Lorenzati M, Tshuva RY, Ferraro A, Balzac F, Turco E, Salvi SU, Myklebust HF, Wang S, Eisenberg J, Chitale M, Girgla NS, Boda E, Reiner O, Buffo A, Di Cunto F, Bielas SL. Pallavicini G, et al. Among authors: lorenzati m. J Clin Invest. 2024 Nov 1;134(21):e175435. doi: 10.1172/JCI175435. J Clin Invest. 2024. PMID: 39316437 Free PMC article.
14 results