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Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1/2 trial interim results.
Sevigny J, Uspenskaya O, Heckman LD, Wong LC, Hatch DA, Tewari A, Vandenberghe R, Irwin DJ, Saracino D, Le Ber I, Ahmed R, Rohrer JD, Boxer AL, Boland S, Sheehan P, Brandes A, Burstein SR, Shykind BM, Kamalakaran S, Daniels CW, David Litwack E, Mahoney E, Velaga J, McNamara I, Sondergaard P, Sajjad SA, Kobayashi YM, Abeliovich A, Hefti F. Sevigny J, et al. Among authors: kobayashi ym. Nat Med. 2024 May;30(5):1406-1415. doi: 10.1038/s41591-024-02973-0. Epub 2024 May 14. Nat Med. 2024. PMID: 38745011 Free PMC article. Clinical Trial.
Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy.
Hathout Y, Brody E, Clemens PR, Cripe L, DeLisle RK, Furlong P, Gordish-Dressman H, Hache L, Henricson E, Hoffman EP, Kobayashi YM, Lorts A, Mah JK, McDonald C, Mehler B, Nelson S, Nikrad M, Singer B, Steele F, Sterling D, Sweeney HL, Williams S, Gold L. Hathout Y, et al. Among authors: kobayashi ym. Proc Natl Acad Sci U S A. 2015 Jun 9;112(23):7153-8. doi: 10.1073/pnas.1507719112. Epub 2015 May 26. Proc Natl Acad Sci U S A. 2015. PMID: 26039989 Free PMC article.
Like-acetylglucosaminyltransferase (LARGE)-dependent modification of dystroglycan at Thr-317/319 is required for laminin binding and arenavirus infection.
Hara Y, Kanagawa M, Kunz S, Yoshida-Moriguchi T, Satz JS, Kobayashi YM, Zhu Z, Burden SJ, Oldstone MB, Campbell KP. Hara Y, et al. Among authors: kobayashi ym. Proc Natl Acad Sci U S A. 2011 Oct 18;108(42):17426-31. doi: 10.1073/pnas.1114836108. Epub 2011 Oct 10. Proc Natl Acad Sci U S A. 2011. PMID: 21987822 Free PMC article.
Sarcolemma-localized nNOS is required to maintain activity after mild exercise.
Kobayashi YM, Rader EP, Crawford RW, Iyengar NK, Thedens DR, Faulkner JA, Parikh SV, Weiss RM, Chamberlain JS, Moore SA, Campbell KP. Kobayashi YM, et al. Nature. 2008 Nov 27;456(7221):511-5. doi: 10.1038/nature07414. Epub 2008 Oct 26. Nature. 2008. PMID: 18953332 Free PMC article.
23 results