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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. Science. 2022 Nov 4;378(6619):523-532. doi: 10.1126/science.abq6656. Epub 2022 Nov 3. Science. 2022. PMID: 36378958 Free PMC article.
A genetically encoded fluorescent sensor for in vivo imaging of GABA.
Marvin JS, Shimoda Y, Magloire V, Leite M, Kawashima T, Jensen TP, Kolb I, Knott EL, Novak O, Podgorski K, Leidenheimer NJ, Rusakov DA, Ahrens MB, Kullmann DM, Looger LL. Marvin JS, et al. Nat Methods. 2019 Aug;16(8):763-770. doi: 10.1038/s41592-019-0471-2. Epub 2019 Jul 15. Nat Methods. 2019. PMID: 31308547
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. Brain. 2024 Feb 1;147(2):542-553. doi: 10.1093/brain/awad387. Brain. 2024. PMID: 38100333 Free PMC article.
Novel therapies for epilepsy in the pipeline.
Mesraoua B, Deleu D, Kullmann DM, Shetty AK, Boon P, Perucca E, Mikati MA, Asadi-Pooya AA. Mesraoua B, et al. Epilepsy Behav. 2019 Aug;97:282-290. doi: 10.1016/j.yebeh.2019.04.042. Epub 2019 Jul 6. Epilepsy Behav. 2019. PMID: 31284159 Review.
Optogenetic and chemogenetic therapies for epilepsy.
Walker MC, Kullmann DM. Walker MC, et al. Neuropharmacology. 2020 May 15;168:107751. doi: 10.1016/j.neuropharm.2019.107751. Epub 2019 Sep 5. Neuropharmacology. 2020. PMID: 31494141 Review.
25 results