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Page 1
The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: Results from trials in healthy adults and patients with spinal muscular atrophy.
Kletzl H, Marquet A, Günther A, Tang W, Heuberger J, Groeneveld GJ, Birkhoff W, Mercuri E, Lochmüller H, Wood C, Fischer D, Gerlach I, Heinig K, Bugawan T, Dziadek S, Kinch R, Czech C, Khwaja O. Kletzl H, et al. Among authors: gerlach i. Neuromuscul Disord. 2019 Jan;29(1):21-29. doi: 10.1016/j.nmd.2018.10.001. Epub 2018 Oct 30. Neuromuscul Disord. 2019. PMID: 30553700 Clinical Trial.
Longitudinal characterization of biomarkers for spinal muscular atrophy.
Bonati U, Holiga Š, Hellbach N, Risterucci C, Bergauer T, Tang W, Hafner P, Thoeni A, Bieri O, Gerlach I, Marquet A, Khwaja O, Sambataro F, Bertolino A, Dukart J, Fischmann A, Fischer D, Czech C. Bonati U, et al. Among authors: gerlach i. Ann Clin Transl Neurol. 2017 Apr 11;4(5):292-304. doi: 10.1002/acn3.406. eCollection 2017 May. Ann Clin Transl Neurol. 2017. PMID: 28491897 Free PMC article.
Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy.
Ratni H, Karp GM, Weetall M, Naryshkin NA, Paushkin SV, Chen KS, McCarthy KD, Qi H, Turpoff A, Woll MG, Zhang X, Zhang N, Yang T, Dakka A, Vazirani P, Zhao X, Pinard E, Green L, David-Pierson P, Tuerck D, Poirier A, Muster W, Kirchner S, Mueller L, Gerlach I, Metzger F. Ratni H, et al. Among authors: gerlach i. J Med Chem. 2016 Jul 14;59(13):6086-100. doi: 10.1021/acs.jmedchem.6b00459. Epub 2016 Jul 6. J Med Chem. 2016. PMID: 27299419
Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.
Naryshkin NA, Weetall M, Dakka A, Narasimhan J, Zhao X, Feng Z, Ling KK, Karp GM, Qi H, Woll MG, Chen G, Zhang N, Gabbeta V, Vazirani P, Bhattacharyya A, Furia B, Risher N, Sheedy J, Kong R, Ma J, Turpoff A, Lee CS, Zhang X, Moon YC, Trifillis P, Welch EM, Colacino JM, Babiak J, Almstead NG, Peltz SW, Eng LA, Chen KS, Mull JL, Lynes MS, Rubin LL, Fontoura P, Santarelli L, Haehnke D, McCarthy KD, Schmucki R, Ebeling M, Sivaramakrishnan M, Ko CP, Paushkin SV, Ratni H, Gerlach I, Ghosh A, Metzger F. Naryshkin NA, et al. Among authors: gerlach i. Science. 2014 Aug 8;345(6197):688-93. doi: 10.1126/science.1250127. Science. 2014. PMID: 25104390
Sembragiline in Moderate Alzheimer's Disease: Results of a Randomized, Double-Blind, Placebo-Controlled Phase II Trial (MAyflOwer RoAD).
Nave S, Doody RS, Boada M, Grimmer T, Savola JM, Delmar P, Pauly-Evers M, Nikolcheva T, Czech C, Borroni E, Ricci B, Dukart J, Mannino M, Carey T, Moran E, Gilaberte I, Muelhardt NM, Gerlach I, Santarelli L, Ostrowitzki S, Fontoura P. Nave S, et al. Among authors: gerlach i. J Alzheimers Dis. 2017;58(4):1217-1228. doi: 10.3233/JAD-161309. J Alzheimers Dis. 2017. PMID: 28550255 Free PMC article. Clinical Trial.
Targeting Huntingtin Expression in Patients with Huntington's Disease.
Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, Wild EJ, Saft C, Barker RA, Blair NF, Craufurd D, Priller J, Rickards H, Rosser A, Kordasiewicz HB, Czech C, Swayze EE, Norris DA, Baumann T, Gerlach I, Schobel SA, Paz E, Smith AV, Bennett CF, Lane RM; Phase 1–2a IONIS-HTTRx Study Site Teams. Tabrizi SJ, et al. Among authors: gerlach i. N Engl J Med. 2019 Jun 13;380(24):2307-2316. doi: 10.1056/NEJMoa1900907. Epub 2019 May 6. N Engl J Med. 2019. PMID: 31059641 Clinical Trial.
Discovery of RO7185876, a Highly Potent γ-Secretase Modulator (GSM) as a Potential Treatment for Alzheimer's Disease.
Ratni H, Alker A, Bartels B, Bissantz C, Chen W, Gerlach I, Limberg A, Lu M, Neidhart W, Pichereau S, Reutlinger M, Rodriguez-Sarmiento RM, Jakob-Roetne R, Schmitt G, Zhang E, Baumann K. Ratni H, et al. Among authors: gerlach i. ACS Med Chem Lett. 2020 Apr 27;11(6):1257-1268. doi: 10.1021/acsmedchemlett.0c00109. eCollection 2020 Jun 11. ACS Med Chem Lett. 2020. PMID: 32551009 Free PMC article.
STIC2 selectively binds ribosome-nascent chain complexes in the cotranslational sorting of Arabidopsis thylakoid proteins.
Stolle DS, Osterhoff L, Treimer P, Lambertz J, Karstens M, Keller JM, Gerlach I, Bischoff A, Dünschede B, Rödiger A, Herrmann C, Baginsky S, Hofmann E, Zoschke R, Armbruster U, Nowaczyk MM, Schünemann D. Stolle DS, et al. Among authors: gerlach i. EMBO J. 2024 Oct;43(20):4699-4719. doi: 10.1038/s44318-024-00211-4. Epub 2024 Aug 27. EMBO J. 2024. PMID: 39192033 Free PMC article.
Eukaryote-specific assembly factor DEAP2 mediates an early step of photosystem II assembly in Arabidopsis.
Keller JM, Frieboes MJ, Jödecke L, Kappel S, Wulff N, Rindfleisch T, Sandoval-Ibanez O, Gerlach I, Thiele W, Bock R, Eirich J, Finkemeier I, Schünemann D, Zoschke R, Schöttler MA, Armbruster U. Keller JM, et al. Among authors: gerlach i. Plant Physiol. 2023 Oct 26;193(3):1970-1986. doi: 10.1093/plphys/kiad446. Plant Physiol. 2023. PMID: 37555435 Free PMC article.
29 results