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In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model.
Chen YA, Kankel MW, Hana S, Lau SK, Zavodszky MI, McKissick O, Mastrangelo N, Dion J, Wang B, Ferretti D, Koske D, Lehman S, Koszka K, McLaughlin H, Liu M, Marshall E, Fabian AJ, Cullen P, Marsh G, Hamann S, Craft M, Sebalusky J, Arnold HM, Driscoll R, Sheehy A, Luo Y, Manca S, Carlile T, Sun C, Sigrist K, McCampbell A, Henderson CE, Lo SC. Chen YA, et al. Among authors: kankel mw. Gene Ther. 2023 May;30(5):443-454. doi: 10.1038/s41434-022-00375-w. Epub 2022 Dec 1. Gene Ther. 2023. PMID: 36450833 Free PMC article.
Poly(GR) and poly(GA) in cerebrospinal fluid as potential biomarkers for C9ORF72-ALS/FTD.
Krishnan G, Raitcheva D, Bartlett D, Prudencio M, McKenna-Yasek DM, Douthwright C, Oskarsson BE, Ladha S, King OD, Barmada SJ, Miller TM, Bowser R, Watts JK, Petrucelli L, Brown RH, Kankel MW, Gao FB. Krishnan G, et al. Among authors: kankel mw. Nat Commun. 2022 May 19;13(1):2799. doi: 10.1038/s41467-022-30387-4. Nat Commun. 2022. PMID: 35589711 Free PMC article.
Antibody Therapy Targeting RAN Proteins Rescues C9 ALS/FTD Phenotypes in C9orf72 Mouse Model.
Nguyen L, Montrasio F, Pattamatta A, Tusi SK, Bardhi O, Meyer KD, Hayes L, Nakamura K, Banez-Coronel M, Coyne A, Guo S, Laboissonniere LA, Gu Y, Narayanan S, Smith B, Nitsch RM, Kankel MW, Rushe M, Rothstein J, Zu T, Grimm J, Ranum LPW. Nguyen L, et al. Among authors: kankel mw. Neuron. 2020 Feb 19;105(4):645-662.e11. doi: 10.1016/j.neuron.2019.11.007. Epub 2019 Dec 9. Neuron. 2020. PMID: 31831332 Free PMC article.
Discovery of Phospholipase D Inhibitors with Improved Drug-like Properties and Central Nervous System Penetrance.
May-Dracka TL, Gao F, Hopkins BT, Hronowski X, Chen T, Chodaparambil JV, Metrick CM, Cullivan M, Enyedy I, Kaliszczak M, Kankel MW, Marx I, Michell-Robinson MA, Murugan P, Kumar PR, Rooney M, Schuman E, Sen A, Wang T, Ye T, Peterson EA. May-Dracka TL, et al. Among authors: kankel mw. ACS Med Chem Lett. 2022 Mar 3;13(4):665-673. doi: 10.1021/acsmedchemlett.1c00682. eCollection 2022 Apr 14. ACS Med Chem Lett. 2022. PMID: 35450377 Free PMC article.
Genetic circuitry of Survival motor neuron, the gene underlying spinal muscular atrophy.
Sen A, Dimlich DN, Guruharsha KG, Kankel MW, Hori K, Yokokura T, Brachat S, Richardson D, Loureiro J, Sivasankaran R, Curtis D, Davidow LS, Rubin LL, Hart AC, Van Vactor D, Artavanis-Tsakonas S. Sen A, et al. Among authors: kankel mw. Proc Natl Acad Sci U S A. 2013 Jun 25;110(26):E2371-80. doi: 10.1073/pnas.1301738110. Epub 2013 Jun 11. Proc Natl Acad Sci U S A. 2013. PMID: 23757500 Free PMC article.
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