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Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism.
Ng J, Barral S, De La Fuente Barrigon C, Lignani G, Erdem FA, Wallings R, Privolizzi R, Rossignoli G, Alrashidi H, Heasman S, Meyer E, Ngoh A, Pope S, Karda R, Perocheau D, Baruteau J, Suff N, Antinao Diaz J, Schorge S, Vowles J, Marshall LR, Cowley SA, Sucic S, Freissmuth M, Counsell JR, Wade-Martins R, Heales SJR, Rahim AA, Bencze M, Waddington SN, Kurian MA. Ng J, et al. Among authors: lignani g. Sci Transl Med. 2021 May 19;13(594):eaaw1564. doi: 10.1126/scitranslmed.aaw1564. Sci Transl Med. 2021. PMID: 34011628 Free PMC article.
Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice.
Counsell JR, Karda R, Diaz JA, Carey L, Wiktorowicz T, Buckley SMK, Ameri S, Ng J, Baruteau J, Almeida F, de Silva R, Simone R, Lugarà E, Lignani G, Lindemann D, Rethwilm A, Rahim AA, Waddington SN, Howe SJ. Counsell JR, et al. Among authors: lignani g. Mol Ther Nucleic Acids. 2018 Sep 7;12:626-634. doi: 10.1016/j.omtn.2018.07.006. Epub 2018 Aug 3. Mol Ther Nucleic Acids. 2018. PMID: 30081233 Free PMC article.
dCas9-Based Scn1a Gene Activation Restores Inhibitory Interneuron Excitability and Attenuates Seizures in Dravet Syndrome Mice.
Colasante G, Lignani G, Brusco S, Di Berardino C, Carpenter J, Giannelli S, Valassina N, Bido S, Ricci R, Castoldi V, Marenna S, Church T, Massimino L, Morabito G, Benfenati F, Schorge S, Leocani L, Kullmann DM, Broccoli V. Colasante G, et al. Among authors: lignani g. Mol Ther. 2020 Jan 8;28(1):235-253. doi: 10.1016/j.ymthe.2019.08.018. Epub 2019 Sep 3. Mol Ther. 2020. PMID: 31607539 Free PMC article.
48 results