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

Year Number of Results
1994 1
2011 1
2013 2
2014 3
2015 1
2016 2
2017 1
2018 1
2019 1
2020 1
2021 3
2022 5
2023 6
2024 1

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25 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: likhite s. 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: likhite s. 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.
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: likhite s. Cell Rep. 2022 Dec 6;41(10):111751. doi: 10.1016/j.celrep.2022.111751. Cell Rep. 2022. PMID: 36476864 Free article.
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: likhite s. 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.
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: likhite s. Front Genet. 2023 Mar 24;14:1118649. doi: 10.3389/fgene.2023.1118649. eCollection 2023. Front Genet. 2023. PMID: 37035740 Free PMC article.
Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease.
Cain JT, Likhite S, White KA, Timm DJ, Davis SS, Johnson TB, Dennys-Rivers CN, Rinaldi F, Motti D, Corcoran S, Morales P, Pierson C, Hughes SM, Lee SY, Kaspar BK, Meyer K, Weimer JM. Cain JT, et al. Among authors: likhite s. Mol Ther. 2019 Oct 2;27(10):1836-1847. doi: 10.1016/j.ymthe.2019.06.015. Epub 2019 Jul 10. Mol Ther. 2019. PMID: 31331814 Free PMC article.
AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models.
Murakami Y, Umeshita S, Imanishi K, Yoshioka Y, Ninomiya A, Sunabori T, Likhite S, Koike M, Meyer KC, Kinoshita T. Murakami Y, et al. Among authors: likhite s. Mol Ther Methods Clin Dev. 2023 Dec 14;32(1):101176. doi: 10.1016/j.omtm.2023.101176. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2023. PMID: 38225934 Free PMC article.
25 results