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Year Number of Results
2011 1
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54 results

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Page 1
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: lignani g. Science. 2022 Nov 4;378(6619):523-532. doi: 10.1126/science.abq6656. Epub 2022 Nov 3. Science. 2022. PMID: 36378958 Free PMC article.
AMPA receptor GluA2 subunit defects are a cause of neurodevelopmental disorders.
Salpietro V, Dixon CL, Guo H, Bello OD, Vandrovcova J, Efthymiou S, Maroofian R, Heimer G, Burglen L, Valence S, Torti E, Hacke M, Rankin J, Tariq H, Colin E, Procaccio V, Striano P, Mankad K, Lieb A, Chen S, Pisani L, Bettencourt C, Männikkö R, Manole A, Brusco A, Grosso E, Ferrero GB, Armstrong-Moron J, Gueden S, Bar-Yosef O, Tzadok M, Monaghan KG, Santiago-Sim T, Person RE, Cho MT, Willaert R, Yoo Y, Chae JH, Quan Y, Wu H, Wang T, Bernier RA, Xia K, Blesson A, Jain M, Motazacker MM, Jaeger B, Schneider AL, Boysen K, Muir AM, Myers CT, Gavrilova RH, Gunderson L, Schultz-Rogers L, Klee EW, Dyment D, Osmond M, Parellada M, Llorente C, Gonzalez-Peñas J, Carracedo A, Van Haeringen A, Ruivenkamp C, Nava C, Heron D, Nardello R, Iacomino M, Minetti C, Skabar A, Fabretto A; SYNAPS Study Group; Raspall-Chaure M, Chez M, Tsai A, Fassi E, Shinawi M, Constantino JN, De Zorzi R, Fortuna S, Kok F, Keren B, Bonneau D, Choi M, Benzeev B, Zara F, Mefford HC, Scheffer IE, Clayton-Smith J, Macaya A, Rothman JE, Eichler EE, Kullmann DM, Houlden H. Salpietro V, et al. Nat Commun. 2019 Jul 12;10(1):3094. doi: 10.1038/s41467-019-10910-w. Nat Commun. 2019. PMID: 31300657 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: lignani g. Brain. 2024 Feb 1;147(2):542-553. doi: 10.1093/brain/awad387. Brain. 2024. PMID: 38100333 Free PMC article.
Neurodevelopmental and synaptic defects in DNAJC6 parkinsonism, amenable to gene therapy.
Abela L, Gianfrancesco L, Tagliatti E, Rossignoli G, Barwick K, Zourray C, Reid KM, Budinger D, Ng J, Counsell J, Simpson A, Pearson TS, Edvardson S, Elpeleg O, Brodsky FM, Lignani G, Barral S, Kurian MA. Abela L, et al. Among authors: lignani g. Brain. 2024 Jun 3;147(6):2023-2037. doi: 10.1093/brain/awae020. Brain. 2024. PMID: 38242634 Free PMC article.
Organoids as a model of status epilepticus.
Street JS, Zourray C, Lignani G. Street JS, et al. Among authors: lignani g. Epilepsy Behav. 2024 Dec;161:110145. doi: 10.1016/j.yebeh.2024.110145. Epub 2024 Nov 17. Epilepsy Behav. 2024. PMID: 39556970 Free article. Review.
Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo.
Kempthorne L, Vaizoglu D, Cammack AJ, Carcolé M, Roberts MJ, Mikheenko A, Fisher A, Suklai P, Muralidharan B, Kroll F, Moens TG, Yshii L, Verschoren S, Hölbling BV, Moreira FC, Katona E, Coneys R, de Oliveira P, Zhang YJ, Jansen K, Daughrity LM, McGown A, Ramesh TM, Van Den Bosch L, Lignani G, Rahim AA, Coyne AN, Petrucelli L, Rihel J, Isaacs AM. Kempthorne L, et al. Among authors: lignani g. Nat Commun. 2025 Jan 8;16(1):459. doi: 10.1038/s41467-024-55550-x. Nat Commun. 2025. PMID: 39779704 Free PMC article.
Epg5 links proteotoxic stress due to defective autophagic clearance and epileptogenesis in Drosophila and Vici syndrome patients.
Deneubourg C, Salimi Dafsari H, Lowe S, Martinez-Cotrina A, Mazaud D, Park SH, Vergani V, Almacellas Barbanoj A, Maroofian R, Averdunk L, Ghayoor-Karimiani E, Jayawant S, Mignot C, Keren B, Peters R, Kamath A, Mattas L, Verma S, Silwal A, Distelmaier F, Houlden H, Lignani G, Antebi A, Jepson J, Jungbluth H, Fanto M. Deneubourg C, et al. Among authors: lignani g. Autophagy. 2025 Feb;21(2):447-459. doi: 10.1080/15548627.2024.2405956. Epub 2024 Oct 10. Autophagy. 2025. PMID: 39342484 Free PMC article.
54 results