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Page 1
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. 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.
On-demand cell-autonomous gene therapy for brain circuit disorders.
Qiu Y, O'Neill N, Maffei B, Zourray C, Almacellas-Barbanoj A, Carpenter JC, Jones SP, Leite M, Turner TJ, Moreira FC, Snowball A, Shekh-Ahmad T, Magloire V, Barral S, Kurian MA, Walker MC, Schorge S, Kullmann DM, Lignani G. Qiu Y, et al. Among authors: carpenter jc. Science. 2022 Nov 4;378(6619):523-532. doi: 10.1126/science.abq6656. Epub 2022 Nov 3. Science. 2022. PMID: 36378958 Free PMC article.
Anti-seizure gene therapy for focal cortical dysplasia.
Almacellas Barbanoj A, Graham RT, Maffei B, Carpenter JC, Leite M, Hoke J, Hardjo F, Scott-Solache J, Chimonides C, Schorge S, Kullmann DM, Magloire V, Lignani G. Almacellas Barbanoj A, et al. Among authors: carpenter jc. Brain. 2024 Feb 1;147(2):542-553. doi: 10.1093/brain/awad387. Brain. 2024. PMID: 38100333 Free PMC article.
Early developmental alterations of CA1 pyramidal cells in Dravet syndrome.
Jones SP, O'Neill N, Carpenter JC, Muggeo S, Colasante G, Kullmann DM, Lignani G. Jones SP, et al. Among authors: carpenter jc. Neurobiol Dis. 2024 Oct 15;201:106688. doi: 10.1016/j.nbd.2024.106688. Epub 2024 Oct 3. Neurobiol Dis. 2024. PMID: 39368670 Free article.
18 results