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Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.
Davidson BL, Gao G, Berry-Kravis E, Bradbury AM, Bönnemann C, Buxbaum JD, Corcoran GR, Gray SJ, Gray-Edwards H, Kleiman RJ, Shaywitz AJ, Wang D, Zoghbi HY, Flotte TR, Tauscher-Wisniewski S, Tifft CJ, Sahin M; Gene Therapy Workshop Faculty. Davidson BL, et al. Among authors: kleiman rj. Mol Ther. 2022 Jul 6;30(7):2416-2428. doi: 10.1016/j.ymthe.2022.05.014. Epub 2022 May 17. Mol Ther. 2022. PMID: 35585789 Free PMC article. Review.
Generation and characterization of human induced pluripotent stem cells (iPSCs) from three male and three female patients with CDKL5 Deficiency Disorder (CDD).
Chen PF, Chen T, Forman TE, Swanson AC, O'Kelly B, Dwyer SA, Buttermore ED, Kleiman R, Js Carrington S, Lavery DJ, Swanson LC, Olson HE, Sahin M. Chen PF, et al. Stem Cell Res. 2021 May;53:102276. doi: 10.1016/j.scr.2021.102276. Epub 2021 Mar 5. Stem Cell Res. 2021. PMID: 33714067 Free PMC article.
Reversal of neurobehavioral social deficits in dystrophic mice using inhibitors of phosphodiesterases PDE5A and PDE9A.
Alexander MS, Gasperini MJ, Tsai PT, Gibbs DE, Spinazzola JM, Marshall JL, Feyder MJ, Pletcher MT, Chekler EL, Morris CA, Sahin M, Harms JF, Schmidt CJ, Kleiman RJ, Kunkel LM. Alexander MS, et al. Among authors: kleiman rj. Transl Psychiatry. 2016 Sep 27;6(9):e901. doi: 10.1038/tp.2016.174. Transl Psychiatry. 2016. PMID: 27676442 Free PMC article.
Endothelial GNAQ p.R183Q Increases ANGPT2 (Angiopoietin-2) and Drives Formation of Enlarged Blood Vessels.
Huang L, Bichsel C, Norris AL, Thorpe J, Pevsner J, Alexandrescu S, Pinto A, Zurakowski D, Kleiman RJ, Sahin M, Greene AK, Bischoff J. Huang L, et al. Among authors: kleiman rj. Arterioscler Thromb Vasc Biol. 2022 Jan;42(1):e27-e43. doi: 10.1161/ATVBAHA.121.316651. Epub 2021 Oct 21. Arterioscler Thromb Vasc Biol. 2022. PMID: 34670408 Free PMC article.
Current Perspectives in Autism Spectrum Disorder: From Genes to Therapy.
Chahrour M, O'Roak BJ, Santini E, Samaco RC, Kleiman RJ, Manzini MC. Chahrour M, et al. Among authors: kleiman rj. J Neurosci. 2016 Nov 9;36(45):11402-11410. doi: 10.1523/JNEUROSCI.2335-16.2016. J Neurosci. 2016. PMID: 27911742 Free PMC article. Review.
Translating genetic and preclinical findings into autism therapies.
Chahrour M, Kleiman RJ, Manzini MC. Chahrour M, et al. Among authors: kleiman rj. Dialogues Clin Neurosci. 2017 Dec;19(4):335-343. doi: 10.31887/DCNS.2017.19.4/cmanzini. Dialogues Clin Neurosci. 2017. PMID: 29398929 Free PMC article. Review.
Therapeutics for cognitive aging.
Shineman DW, Salthouse TA, Launer LJ, Hof PR, Bartzokis G, Kleiman R, Luine V, Buccafusco JJ, Small GW, Aisen PS, Lowe DA, Fillit HM. Shineman DW, et al. Ann N Y Acad Sci. 2010 Apr;1191 Suppl 1(Suppl 1):E1-15. doi: 10.1111/j.1749-6632.2010.05532.x. Ann N Y Acad Sci. 2010. PMID: 20392284 Free PMC article.
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