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TGS1 impacts snRNA 3'-end processing, ameliorates survival motor neuron-dependent neurological phenotypes in vivo and prevents neurodegeneration.
Chen L, Roake CM, Maccallini P, Bavasso F, Dehghannasiri R, Santonicola P, Mendoza-Ferreira N, Scatolini L, Rizzuti L, Esposito A, Gallotta I, Francia S, Cacchione S, Galati A, Palumbo V, Kobin MA, Tartaglia GG, Colantoni A, Proietti G, Wu Y, Hammerschmidt M, De Pittà C, Sales G, Salzman J, Pellizzoni L, Wirth B, Di Schiavi E, Gatti M, Artandi SE, Raffa GD. Chen L, et al. Nucleic Acids Res. 2022 Nov 28;50(21):12400-12424. doi: 10.1093/nar/gkac659. Nucleic Acids Res. 2022. PMID: 35947650 Free PMC article.
Disruption of Telomerase RNA Maturation Kinetics Precipitates Disease.
Roake CM, Chen L, Chakravarthy AL, Ferrell JE Jr, Raffa GD, Artandi SE. Roake CM, et al. Among authors: chen l. Mol Cell. 2019 May 16;74(4):688-700.e3. doi: 10.1016/j.molcel.2019.02.033. Epub 2019 Mar 28. Mol Cell. 2019. PMID: 30930056 Free PMC article.
The S-adenosylmethionine analog sinefungin inhibits the trimethylguanosine synthase TGS1 to promote telomerase activity and telomere lengthening.
Galati A, Scatolini L, Micheli E, Bavasso F, Cicconi A, Maccallini P, Chen L, Roake CM, Schoeftner S, Artandi SE, Gatti M, Cacchione S, Raffa GD. Galati A, et al. Among authors: chen l. FEBS Lett. 2022 Jan;596(1):42-52. doi: 10.1002/1873-3468.14240. Epub 2021 Dec 5. FEBS Lett. 2022. PMID: 34817067 Free article.
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus.
Vaidyanathan S, Baik R, Chen L, Bravo DT, Suarez CJ, Abazari SM, Salahudeen AA, Dudek AM, Teran CA, Davis TH, Lee CM, Bao G, Randell SH, Artandi SE, Wine JJ, Kuo CJ, Desai TJ, Nayak JV, Sellers ZM, Porteus MH. Vaidyanathan S, et al. Among authors: chen l. Mol Ther. 2022 Jan 5;30(1):223-237. doi: 10.1016/j.ymthe.2021.03.023. Epub 2021 Mar 29. Mol Ther. 2022. PMID: 33794364 Free PMC article.
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