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Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.
Ellison S, Buckland K, Learmonth Y, Day V, Kalra S, Howe L, Roman-Rodriguez FJ, Bonafont J, Booth L, Holley R, Smythe J, Jones S, Thrasher A, Booth C, Bigger BW. Ellison S, et al. Among authors: bigger bw. Mol Ther Methods Clin Dev. 2024 May 21;32(2):101271. doi: 10.1016/j.omtm.2024.101271. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38946936 Free PMC article.
Collagen fibril formation at the plasma membrane occurs independently from collagen secretion.
Pickard A, Garva R, Adamson A, Calverley BC, Hoyle A, Hayward CE, Spiller D, Lu Y, Hodson N, Mandolfo O, Kim KK, Bou-Gharios G, Swift J, Bigger B, Kadler KE. Pickard A, et al. Among authors: bigger b. bioRxiv [Preprint]. 2024 May 10:2024.05.09.593302. doi: 10.1101/2024.05.09.593302. bioRxiv. 2024. PMID: 38766096 Free PMC article. Preprint.
Secondary deficiency of neuraminidase 1 contributes to CNS pathology in neurological mucopolysaccharidoses via hypersialylation of brain glycoproteins.
Xu T, Heon-Roberts R, Moore T, Dubot P, Pan X, Guo T, Cairo CW, Holley R, Bigger B, Durcan TM, Levade T, Ausseil J, Amilhon B, Gorelik A, Nagar B, Sturiale L, Palmigiano A, Röckle I, Thiesler H, Hildebrandt H, Garozzo D, Pshezhetsky AV. Xu T, et al. Among authors: bigger b. bioRxiv [Preprint]. 2024 Apr 27:2024.04.26.587986. doi: 10.1101/2024.04.26.587986. bioRxiv. 2024. PMID: 38712143 Free PMC article. Preprint.
Improving newborn screening test performance for metachromatic leukodystrophy: Recommendation from a pre-pilot study that identified a late-infantile case for treatment.
Wu THY, Brown HA, Church HJ, Kershaw CJ, Hutton R, Egerton C, Cooper J, Tylee K, Cohen RN, Gokhale D, Ram D, Morton G, Henderson M, Bigger BW, Jones SA. Wu THY, et al. Among authors: bigger bw. Mol Genet Metab. 2024 May;142(1):108349. doi: 10.1016/j.ymgme.2024.108349. Epub 2024 Feb 20. Mol Genet Metab. 2024. PMID: 38458124 Free article.
112 results