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mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria.
Gurung S, Timmermand OV, Perocheau D, Gil-Martinez AL, Minnion M, Touramanidou L, Fang S, Messina M, Khalil Y, Spiewak J, Barber AR, Edwards RS, Pinto PL, Finn PF, Cavedon A, Siddiqui S, Rice L, Martini PGV, Ridout D, Heywood W, Hargreaves I, Heales S, Mills PB, Waddington SN, Gissen P, Eaton S, Ryten M, Feelisch M, Frassetto A, Witney TH, Baruteau J. Gurung S, et al. Among authors: spiewak j. Sci Transl Med. 2024 Jan 10;16(729):eadh1334. doi: 10.1126/scitranslmed.adh1334. Epub 2024 Jan 10. Sci Transl Med. 2024. PMID: 38198573 Free PMC article.
Niemann-Pick type C disease as proof-of-concept for intelligent biomarker panel selection in neurometabolic disorders.
Papandreou A, Doykov I, Spiewak J, Komarov N, Habermann S, Kurian MA, Mills PB, Mills K, Gissen P, Heywood WE; Clinical cohort recruitment and characterization group. Papandreou A, et al. Among authors: spiewak j. Dev Med Child Neurol. 2022 Dec;64(12):1539-1546. doi: 10.1111/dmcn.15334. Epub 2022 Jul 14. Dev Med Child Neurol. 2022. PMID: 35833379 Free PMC article.
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease.
Jeyakumar JM, Kia A, Tam LCS, McIntosh J, Spiewak J, Mills K, Heywood W, Chisari E, Castaldo N, Verhoef D, Hosseini P, Kalcheva P, Cocita C, Miranda CJ, Canavese M, Khinder J, Rosales C, Hughes D, Sheridan R, Corbau R, Nathwani A. Jeyakumar JM, et al. Among authors: spiewak j. Gene Ther. 2023 Jun;30(6):487-502. doi: 10.1038/s41434-022-00381-y. Epub 2023 Jan 11. Gene Ther. 2023. PMID: 36631545 Free PMC article.
Operation Moonshot: rapid translation of a SARS-CoV-2 targeted peptide immunoaffinity liquid chromatography-tandem mass spectrometry test from research into routine clinical use.
Hällqvist J, Lane D, Shapanis A, Davis K, Heywood WE, Doykov I, Śpiewak J, Ghansah N, Keevil B, Gupta P, Jukes-Jones R, Singh R, Foley D, Vissers JPC, Pattison R, Ferries S, Wardle R, Bartlett A, Calton LJ, Anderson L, Razavi M, Pearson T, Pope M, Yip R, Ng LL, Nicholas BI, Bailey A, Noel D, Dalton RN, Heales S, Hopley C, Pitt AR, Barran P, Jones DJL, Mills K, Skipp P, Carling RS. Hällqvist J, et al. Among authors: spiewak j. Clin Chem Lab Med. 2022 Nov 17;61(2):302-310. doi: 10.1515/cclm-2022-1000. Print 2023 Jan 27. Clin Chem Lab Med. 2022. PMID: 36395058 Free article.
Plasma proteomic signature predicts who will get persistent symptoms following SARS-CoV-2 infection.
Captur G, Moon JC, Topriceanu CC, Joy G, Swadling L, Hallqvist J, Doykov I, Patel N, Spiewak J, Baldwin T, Hamblin M, Menacho K, Fontana M, Treibel TA, Manisty C, O'Brien B, Gibbons JM, Pade C, Brooks T, Altmann DM, Boyton RJ, McKnight Á, Maini MK, Noursadeghi M, Mills K, Heywood WE; UK COVIDsortium Investigators. Captur G, et al. Among authors: spiewak j. EBioMedicine. 2022 Nov;85:104293. doi: 10.1016/j.ebiom.2022.104293. Epub 2022 Sep 28. EBioMedicine. 2022. PMID: 36182629 Free PMC article.
Quantitative, multiplexed, targeted proteomics for ascertaining variant specific SARS-CoV-2 antibody response.
Doykov I, Baldwin T, Spiewak J, Gilmour KC, Gibbons JM, Pade C, Reynolds CJ, Áine McKnight, Noursadeghi M, Maini MK, Manisty C, Treibel T, Captur G, Fontana M, Boyton RJ, Altmann DM, Brooks T, Semper A; UK COVIDsortium Investigators; Moon JC, Kevin Mills, Heywood WE. Doykov I, et al. Among authors: spiewak j. Cell Rep Methods. 2022 Sep 19;2(9):100279. doi: 10.1016/j.crmeth.2022.100279. Epub 2022 Aug 12. Cell Rep Methods. 2022. PMID: 35975199 Free PMC article.
15 results