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A highly efficient transgene knock-in technology in clinically relevant cell types.
Allen AG, Khan SQ, Margulies CM, Viswanathan R, Lele S, Blaha L, Scott SN, Izzo KM, Gerew A, Pattali R, Cochran NR, Holland CS, Zhao AH, Sherman SE, Jaskolka MC, Wu M, Wilson AC, Sun X, Ciulla DM, Zhang D, Nelson JD, Zhang P, Mazzucato P, Huang Y, Giannoukos G, Marco E, Nehil M, Follit JA, Chang KH, Shearman MS, Wilson CJ, Zuris JA. Allen AG, et al. Among authors: shearman ms. Nat Biotechnol. 2024 Mar;42(3):458-469. doi: 10.1038/s41587-023-01779-8. Epub 2023 May 1. Nat Biotechnol. 2024. PMID: 37127662
The Impact of Inherited Retinal Diseases in the United States of America (US) and Canada from a Cost-of-Illness Perspective.
Gong J, Cheung S, Fasso-Opie A, Galvin O, Moniz LS, Earle D, Durham T, Menzo J, Li N, Duffy S, Dolgin J, Shearman MS, Fiorani C, Banhazi J, Daly A. Gong J, et al. Among authors: shearman ms. Clin Ophthalmol. 2021 Jul 1;15:2855-2866. doi: 10.2147/OPTH.S313719. eCollection 2021. Clin Ophthalmol. 2021. PMID: 34234408 Free PMC article.
Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy.
Song C, Dufour VL, Cideciyan AV, Ye GJ, Swider M, Newmark JA, Timmers AM, Robinson PM, Knop DR, Chulay JD, Jacobson SG, Aguirre GD, Beltran WA, Shearman MS. Song C, et al. Among authors: shearman ms. Hum Gene Ther. 2020 Jul;31(13-14):743-755. doi: 10.1089/hum.2019.337. Epub 2020 Jun 29. Hum Gene Ther. 2020. PMID: 32414297 Free PMC article.
Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa.
Dufour VL, Cideciyan AV, Ye GJ, Song C, Timmers A, Habecker PL, Pan W, Weinstein NM, Swider M, Durham AC, Ying GS, Robinson PM, Jacobson SG, Knop DR, Chulay JD, Shearman MS, Aguirre GD, Beltran WA. Dufour VL, et al. Among authors: shearman ms. Hum Gene Ther. 2020 Feb;31(3-4):253-267. doi: 10.1089/hum.2019.297. Epub 2020 Feb 6. Hum Gene Ther. 2020. PMID: 31910043 Free PMC article.
Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice.
Song C, Conlon TJ, Deng WT, Coleman KE, Zhu P, Plummer C, Mandapati S, Van Hoosear M, Green KB, Sonnentag P, Sharma AK, Timmers A, Robinson PM, Knop DR, Hauswirth WW, Chulay JD, Shearman MS, Ye GJ. Song C, et al. Among authors: shearman ms. Hum Gene Ther Clin Dev. 2018 Dec;29(4):188-197. doi: 10.1089/humc.2018.168. Hum Gene Ther Clin Dev. 2018. PMID: 30280954 Free PMC article.
Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.
Beltran WA, Cideciyan AV, Boye SE, Ye GJ, Iwabe S, Dufour VL, Marinho LF, Swider M, Kosyk MS, Sha J, Boye SL, Peterson JJ, Witherspoon CD, Alexander JJ, Ying GS, Shearman MS, Chulay JD, Hauswirth WW, Gamlin PD, Jacobson SG, Aguirre GD. Beltran WA, et al. Among authors: shearman ms. Mol Ther. 2017 Aug 2;25(8):1866-1880. doi: 10.1016/j.ymthe.2017.05.004. Epub 2017 May 27. Mol Ther. 2017. PMID: 28566226 Free PMC article.
Safety and Efficacy Evaluation of rAAV2tYF-PR1.7-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep.
Gootwine E, Ofri R, Banin E, Obolensky A, Averbukh E, Ezra-Elia R, Ross M, Honig H, Rosov A, Yamin E, Ye GJ, Knop DR, Robinson PM, Chulay JD, Shearman MS. Gootwine E, et al. Among authors: shearman ms. Hum Gene Ther Clin Dev. 2017 Jun;28(2):96-107. doi: 10.1089/humc.2017.028. Epub 2017 May 5. Hum Gene Ther Clin Dev. 2017. PMID: 28478700
Discovery of novel triazolobenzazepinones as γ-secretase modulators with central Aβ42 lowering in rodents and rhesus monkeys.
Fischer C, Zultanski SL, Zhou H, Methot JL, Shah S, Hayashi I, Hughes BL, Moxham CM, Bays NW, Smotrov N, Hill AD, Pan BS, Wu Z, Moy LY, Tanga F, Kenific C, Cruz JC, Walker D, Bouthillette M, Nikov GN, Deshmukh SV, Jeliazkova-Mecheva VV, Diaz D, Michener MS, Cook JJ, Munoz B, Shearman MS. Fischer C, et al. Among authors: shearman ms. Bioorg Med Chem Lett. 2015 Sep 1;25(17):3488-94. doi: 10.1016/j.bmcl.2015.07.003. Epub 2015 Jul 9. Bioorg Med Chem Lett. 2015. PMID: 26212776
Potent benzoazepinone γ-secretase modulators with improved bioavailability.
Methot JL, Fischer C, Li C, Rivkin A, Ahearn SP, Brown WC, Kattar S, Kelley E, Mampreian DM, Schell A, Rosenau A, Zhou H, Ball R, Deshmukh SV, Jeliazkova-Mecheva VV, Diaz D, Moy LY, Kenific CM, Moxham C, Shah S, Nuthall H, Szewczak AA, Hill A, Hughes B, Smotrov N, Munoz B, Miller TA, Shearman MS. Methot JL, et al. Among authors: shearman ms. Bioorg Med Chem Lett. 2015 Sep 1;25(17):3495-500. doi: 10.1016/j.bmcl.2015.06.032. Epub 2015 Jun 15. Bioorg Med Chem Lett. 2015. PMID: 26142947
102 results