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Page 1
Development of Assays to Measure GNE Gene Potency and Gene Replacement in Skeletal Muscle.
J Neuromuscul Dis. 2023;10(5):797-812. doi: 10.3233/JND-221596.
J Neuromuscul Dis. 2023.
PMID: 37458043
Free PMC article.
Therapeutic efficacy of rscAAVrh74.miniCMV.LIPA gene therapy in a mouse model of lysosomal acid lipase deficiency.
Lam P, Ashbrook A, Zygmunt DA, Yan C, Du H, Martin PT.
Lam P, et al. Among authors: ashbrook a.
Mol Ther Methods Clin Dev. 2022 Aug 4;26:413-426. doi: 10.1016/j.omtm.2022.08.001. eCollection 2022 Sep 8.
Mol Ther Methods Clin Dev. 2022.
PMID: 36092360
Free PMC article.
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rAAVrh74.MCK.GALGT2 Demonstrates Safety and Widespread Muscle Glycosylation after Intravenous Delivery in C57BL/6J Mice.
Zygmunt DA, Xu R, Jia Y, Ashbrook A, Menke C, Shao G, Yoon JH, Hamilton S, Pisharath H, Bolon B, Martin PT.
Zygmunt DA, et al. Among authors: ashbrook a.
Mol Ther Methods Clin Dev. 2019 Oct 21;15:305-319. doi: 10.1016/j.omtm.2019.10.005. eCollection 2019 Dec 13.
Mol Ther Methods Clin Dev. 2019.
PMID: 31890730
Free PMC article.
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Serum Antibodies to N-Glycolylneuraminic Acid Are Elevated in Duchenne Muscular Dystrophy and Correlate with Increased Disease Pathology in Cmah-/-mdx Mice.
Martin PT, Kawanishi K, Ashbrook A, Golden B, Samraj A, Crowe KE, Zygmunt DA, Okerblom J, Yu H, Maki A, Diaz S, Chen X, Janssen PML, Varki A.
Martin PT, et al. Among authors: ashbrook a.
Am J Pathol. 2021 Aug;191(8):1474-1486. doi: 10.1016/j.ajpath.2021.04.015.
Am J Pathol. 2021.
PMID: 34294193
Free PMC article.
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Short-term treatment of golden retriever muscular dystrophy (GRMD) dogs with rAAVrh74.MHCK7.GALGT2 induces muscle glycosylation and utrophin expression but has no significant effect on muscle strength.
Martin PT, Zygmunt DA, Ashbrook A, Hamilton S, Packer D, Birch SM, Bettis AK, Balog-Alvarez CJ, Guo LJ, Nghiem PP, Kornegay JN.
Martin PT, et al. Among authors: ashbrook a.
PLoS One. 2021 Mar 26;16(3):e0248721. doi: 10.1371/journal.pone.0248721. eCollection 2021.
PLoS One. 2021.
PMID: 33770101
Free PMC article.
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