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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
2004 1
2007 2
2008 3
2009 2
2010 2
2011 2
2012 2
2013 3
2014 5
2015 3
2016 4
2017 4
2018 3
2019 1
2020 3
2021 11
2022 13
2023 9
2024 3

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66 results

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Page 1
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK. Mendell JR, et al. Among authors: meyer k. N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198. N Engl J Med. 2017. PMID: 29091557 Free article. Clinical Trial.
Optimization and validation of CAR transduction into human primary NK cells using CRISPR and AAV.
Naeimi Kararoudi M, Likhite S, Elmas E, Yamamoto K, Schwartz M, Sorathia K, de Souza Fernandes Pereira M, Sezgin Y, Devine RD, Lyberger JM, Behbehani GK, Chakravarti N, Moriarity BS, Meyer K, Lee DA. Naeimi Kararoudi M, et al. Among authors: meyer k. Cell Rep Methods. 2022 Jun 13;2(6):100236. doi: 10.1016/j.crmeth.2022.100236. eCollection 2022 Jun 20. Cell Rep Methods. 2022. PMID: 35784645 Free PMC article.
Opportunities for mitochondrial disease gene therapy.
Viscomi C, van den Ameele J, Meyer KC, Chinnery PF. Viscomi C, et al. Among authors: meyer kc. Nat Rev Drug Discov. 2023 Jun;22(6):429-430. doi: 10.1038/d41573-023-00067-z. Nat Rev Drug Discov. 2023. PMID: 37106085 No abstract available.
Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy.
Sinha Ray S, Dutta D, Dennys C, Powers S, Roussel F, Lisowski P, Glažar P, Zhang X, Biswas P, Caporale JR, Rajewsky N, Bickle M, Wein N, Bellen HJ, Likhite S, Marcogliese PC, Meyer KC. Sinha Ray S, et al. Among authors: meyer kc. Cell Rep. 2022 Dec 6;41(10):111751. doi: 10.1016/j.celrep.2022.111751. Cell Rep. 2022. PMID: 36476864 Free article.
Editorial: Personalized medicine for neuromuscular disorders.
Bartoli M, Bailey RM, Meyer K, Barthélémy F. Bartoli M, et al. Among authors: meyer k. Front Cell Dev Biol. 2023 Nov 16;11:1329048. doi: 10.3389/fcell.2023.1329048. eCollection 2023. Front Cell Dev Biol. 2023. PMID: 38033860 Free PMC article. No abstract available.
Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates.
Powers S, Likhite S, Gadalla KK, Miranda CJ, Huffenberger AJ, Dennys C, Foust KD, Morales P, Pierson CR, Rinaldi F, Perry S, Bolon B, Wein N, Cobb S, Kaspar BK, Meyer KC. Powers S, et al. Among authors: meyer kc. Mol Ther. 2023 Sep 6;31(9):2767-2782. doi: 10.1016/j.ymthe.2023.07.013. Epub 2023 Jul 22. Mol Ther. 2023. PMID: 37481701 Free article.
Intellectual disability: dendritic anomalies and emerging genetic perspectives.
Quach TT, Stratton HJ, Khanna R, Kolattukudy PE, Honnorat J, Meyer K, Duchemin AM. Quach TT, et al. Among authors: meyer k. Acta Neuropathol. 2021 Feb;141(2):139-158. doi: 10.1007/s00401-020-02244-5. Epub 2020 Nov 23. Acta Neuropathol. 2021. PMID: 33226471 Free PMC article. Review.
Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease.
Johnson TB, Brudvig JJ, Likhite S, Pratt MA, White KA, Cain JT, Booth CD, Timm DJ, Davis SS, Meyerink B, Pineda R, Dennys-Rivers C, Kaspar BK, Meyer K, Weimer JM. Johnson TB, et al. Among authors: meyer k. Front Genet. 2023 Mar 24;14:1118649. doi: 10.3389/fgene.2023.1118649. eCollection 2023. Front Genet. 2023. PMID: 37035740 Free PMC article.
PolyGA targets the ER stress-adaptive response by impairing GRP75 function at the MAM in C9ORF72-ALS/FTD.
Pilotto F, Schmitz A, Maharjan N, Diab R, Odriozola A, Tripathi P, Yamoah A, Scheidegger O, Oestmann A, Dennys CN, Sinha Ray S, Rodrigo R, Kolb S, Aronica E, Di Santo S, Widmer HR, Charlet-Berguerand N, Selvaraj BT, Chandran S, Meyer K, Zuber B, Goswami A, Weis J, Saxena S. Pilotto F, et al. Among authors: meyer k. Acta Neuropathol. 2022 Nov;144(5):939-966. doi: 10.1007/s00401-022-02494-5. Epub 2022 Sep 19. Acta Neuropathol. 2022. PMID: 36121477 Free PMC article.
66 results