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Page 1
Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines.
Wei SC, Cantor AJ, Walleshauser J, Mepani R, Melton K, Bans A, Khekare P, Gupta S, Wang J, Soares C, Kiwan R, Lee J, McCawley S, Jani V, Leong WI, Shahi PK, Chan J, Boivin P, Otoupal P, Pattnaik BR, Gamm DM, Saha K, Gowen BG, Haak-Frendscho M, Janatpour MJ, Silverman AP. Wei SC, et al. Among authors: silverman ap. bioRxiv [Preprint]. 2024 Dec 30:2024.12.30.630799. doi: 10.1101/2024.12.30.630799. bioRxiv. 2024. PMID: 39803585 Free PMC article. Preprint.
Identification of a Guide RNA Targeting an Ultraconserved Element for Evaluation of Cas9 Genome Editors Across Mammalian Species.
Gowen BG, Khekare P, McCawley SR, Melton K, Soares C, Chan J, Jani V, Boivin P, Bans A, Leong WI, Cantor AJ, Walleshauser J, Otoupal PB, Mepani RJ, Silverman AP, Haak-Frendscho M, Wei SC. Gowen BG, et al. Among authors: silverman ap. CRISPR J. 2024 Dec;7(6):306-309. doi: 10.1089/crispr.2024.0053. Epub 2024 Sep 23. CRISPR J. 2024. PMID: 39308435 No abstract available.
Identification of bacterial protease domains that cleave human IgM.
Windgassen T, Kruse N, Ferrer B, Du F, Kumar H, Silverman AP. Windgassen T, et al. Among authors: silverman ap. Enzyme Microb Technol. 2024 Feb;173:110366. doi: 10.1016/j.enzmictec.2023.110366. Epub 2023 Nov 25. Enzyme Microb Technol. 2024. PMID: 38061198 Free article.
Oral enzyme therapy for maple syrup urine disease (MSUD) suppresses plasma leucine levels in intermediate MSUD mice and healthy nonhuman primates.
Skvorak K, Liu J, Kruse N, Mehmood R, Das S, Jenne S, Chng C, Lao UL, Duan D, Asfaha J, Du F, Teadt L, Sero A, Ching C, Riggins J, Pope L, Yan P, Mashiana H, Ismaili MHA, McCluskie K, Huisman G, Silverman AP. Skvorak K, et al. Among authors: silverman ap. J Inherit Metab Dis. 2023 Nov;46(6):1089-1103. doi: 10.1002/jimd.12662. Epub 2023 Aug 9. J Inherit Metab Dis. 2023. PMID: 37494004
An orally administered enzyme therapeutic for homocystinuria that suppresses homocysteine by metabolizing methionine in the gastrointestinal tract.
Skvorak K, Mitchell V, Teadt L, Franklin KA, Lee HO, Kruse N, Huitt-Roehl C, Hang J, Du F, Galanie S, Guan S, Aijaz H, Zhang N, Rajkovic G, Kruger WD, Ismaili MHA, Huisman G, McCluskie K, Silverman AP. Skvorak K, et al. Among authors: silverman ap. Mol Genet Metab. 2023 Aug;139(4):107653. doi: 10.1016/j.ymgme.2023.107653. Epub 2023 Jul 12. Mol Genet Metab. 2023. PMID: 37463544 Free article.
Optimizing human α-galactosidase for treatment of Fabry disease.
Hallows WC, Skvorak K, Agard N, Kruse N, Zhang X, Zhu Y, Botham RC, Chng C, Shukla C, Lao J, Miller M, Sero A, Viduya J, Ismaili MHA, McCluskie K, Schiffmann R, Silverman AP, Shen JS, Huisman GW. Hallows WC, et al. Among authors: silverman ap. Sci Rep. 2023 Mar 23;13(1):4748. doi: 10.1038/s41598-023-31777-4. Sci Rep. 2023. PMID: 36959353 Free PMC article.
Brain delivery of therapeutic proteins using an Fc fragment blood-brain barrier transport vehicle in mice and monkeys.
Kariolis MS, Wells RC, Getz JA, Kwan W, Mahon CS, Tong R, Kim DJ, Srivastava A, Bedard C, Henne KR, Giese T, Assimon VA, Chen X, Zhang Y, Solanoy H, Jenkins K, Sanchez PE, Kane L, Miyamoto T, Chew KS, Pizzo ME, Liang N, Calvert MEK, DeVos SL, Baskaran S, Hall S, Sweeney ZK, Thorne RG, Watts RJ, Dennis MS, Silverman AP, Zuchero YJY. Kariolis MS, et al. Among authors: silverman ap. Sci Transl Med. 2020 May 27;12(545):eaay1359. doi: 10.1126/scitranslmed.aay1359. Sci Transl Med. 2020. PMID: 32461332
Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice.
Ullman JC, Arguello A, Getz JA, Bhalla A, Mahon CS, Wang J, Giese T, Bedard C, Kim DJ, Blumenfeld JR, Liang N, Ravi R, Nugent AA, Davis SS, Ha C, Duque J, Tran HL, Wells RC, Lianoglou S, Daryani VM, Kwan W, Solanoy H, Nguyen H, Earr T, Dugas JC, Tuck MD, Harvey JL, Reyzer ML, Caprioli RM, Hall S, Poda S, Sanchez PE, Dennis MS, Gunasekaran K, Srivastava A, Sandmann T, Henne KR, Thorne RG, Di Paolo G, Astarita G, Diaz D, Silverman AP, Watts RJ, Sweeney ZK, Kariolis MS, Henry AG. Ullman JC, et al. Among authors: silverman ap. Sci Transl Med. 2020 May 27;12(545):eaay1163. doi: 10.1126/scitranslmed.aay1163. Sci Transl Med. 2020. PMID: 32461331
Targeting of nonlipidated, aggregated apoE with antibodies inhibits amyloid accumulation.
Liao F, Li A, Xiong M, Bien-Ly N, Jiang H, Zhang Y, Finn MB, Hoyle R, Keyser J, Lefton KB, Robinson GO, Serrano JR, Silverman AP, Guo JL, Getz J, Henne K, Leyns CE, Gallardo G, Ulrich JD, Sullivan PM, Lerner EP, Hudry E, Sweeney ZK, Dennis MS, Hyman BT, Watts RJ, Holtzman DM. Liao F, et al. Among authors: silverman ap. J Clin Invest. 2018 May 1;128(5):2144-2155. doi: 10.1172/JCI96429. Epub 2018 Mar 30. J Clin Invest. 2018. PMID: 29600961 Free PMC article.
47 results