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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: schorge s. Sci Transl Med. 2021 May 19;13(594):eaaw1564. doi: 10.1126/scitranslmed.aaw1564. Sci Transl Med. 2021. PMID: 34011628 Free PMC article.
Mutations in SLC12A5 in epilepsy of infancy with migrating focal seizures.
Stödberg T, McTague A, Ruiz AJ, Hirata H, Zhen J, Long P, Farabella I, Meyer E, Kawahara A, Vassallo G, Stivaros SM, Bjursell MK, Stranneheim H, Tigerschiöld S, Persson B, Bangash I, Das K, Hughes D, Lesko N, Lundeberg J, Scott RC, Poduri A, Scheffer IE, Smith H, Gissen P, Schorge S, Reith ME, Topf M, Kullmann DM, Harvey RJ, Wedell A, Kurian MA. Stödberg T, et al. Among authors: schorge s. Nat Commun. 2015 Sep 3;6:8038. doi: 10.1038/ncomms9038. Nat Commun. 2015. PMID: 26333769 Free PMC article.
Biochemical autoregulatory gene therapy for focal epilepsy.
Lieb A, Qiu Y, Dixon CL, Heller JP, Walker MC, Schorge S, Kullmann DM. Lieb A, et al. Among authors: schorge s. Nat Med. 2018 Sep;24(9):1324-1329. doi: 10.1038/s41591-018-0103-x. Epub 2018 Jul 9. Nat Med. 2018. PMID: 29988123 Free PMC article.
Epilepsy Gene Therapy Using an Engineered Potassium Channel.
Snowball A, Chabrol E, Wykes RC, Shekh-Ahmad T, Cornford JH, Lieb A, Hughes MP, Massaro G, Rahim AA, Hashemi KS, Kullmann DM, Walker MC, Schorge S. Snowball A, et al. Among authors: schorge s. J Neurosci. 2019 Apr 17;39(16):3159-3169. doi: 10.1523/JNEUROSCI.1143-18.2019. Epub 2019 Feb 12. J Neurosci. 2019. PMID: 30755487 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: schorge s. 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.
76 results