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Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial.
Lawlor MW, Schoser B, Margeta M, Sewry CA, Jones KA, Shieh PB, Kuntz NL, Smith BK, Dowling JJ, Müller-Felber W, Bönnemann CG, Seferian AM, Blaschek A, Neuhaus S, Foley AR, Saade DN, Tsuchiya E, Qasim UR, Beatka M, Prom MJ, Ott E, Danielson S, Krakau P, Kumar SN, Meng H, Vanden Avond M, Wells C, Gordish-Dressman H, Beggs AH, Christensen S, Conner E, James ES, Lee J, Sadhu C, Miller W, Sepulveda B, Varfaj F, Prasad S, Rico S. Lawlor MW, et al. Among authors: vanden avond m. EBioMedicine. 2024 Jan;99:104894. doi: 10.1016/j.ebiom.2023.104894. Epub 2023 Dec 12. EBioMedicine. 2024. PMID: 38086156 Free PMC article.
Aberrations in Energetic Metabolism and Stress-Related Pathways Contribute to Pathophysiology in the Neb Conditional Knockout Mouse Model of Nemaline Myopathy.
Slick RA, Tinklenberg JA, Sutton J, Zhang L, Meng H, Beatka MJ, Vanden Avond M, Prom MJ, Ott E, Montanaro F, Heisner J, Toro R, Granzier H, Geurts AM, Stowe DF, Hill RB, Lawlor MW. Slick RA, et al. Among authors: vanden avond m. Am J Pathol. 2023 Oct;193(10):1528-1547. doi: 10.1016/j.ajpath.2023.06.009. Epub 2023 Jul 6. Am J Pathol. 2023. PMID: 37422147
Different Mouse Models of Nemaline Myopathy Harboring Acta1 Mutations Display Differing Abnormalities Related to Mitochondrial Biology.
Tinklenberg JA, Slick RA, Sutton J, Zhang L, Meng H, Beatka MJ, Vanden Avond M, Prom MJ, Ott E, Montanaro F, Heisner J, Toro R, Hardeman EC, Geurts AM, Stowe DF, Hill RB, Lawlor MW. Tinklenberg JA, et al. Among authors: vanden avond m. Am J Pathol. 2023 Oct;193(10):1548-1567. doi: 10.1016/j.ajpath.2023.06.008. Epub 2023 Jul 5. Am J Pathol. 2023. PMID: 37419385
Erratum: Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice.
Hamm SE, Fathalikhani DD, Bukovec KE, Addington AK, Zhang H, Perry JB, McMillan RP, Lawlor MW, Prom MJ, Vanden Avond MA, Kumar SN, Coleman KE, Dupont JB, Mack DL, Brown DA, Morris CA, Gonzalez JP, Grange RW. Hamm SE, et al. Among authors: vanden avond ma. Mol Ther Methods Clin Dev. 2021 Nov 5;23:460. doi: 10.1016/j.omtm.2021.10.005. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34820472 Free PMC article.
Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice.
Hamm SE, Fathalikhani DD, Bukovec KE, Addington AK, Zhang H, Perry JB, McMillan RP, Lawlor MW, Prom MJ, Vanden Avond MA, Kumar SN, Coleman KE, Dupont JB, Mack DL, Brown DA, Morris CA, Gonzalez JP, Grange RW. Hamm SE, et al. Among authors: vanden avond ma. Mol Ther Methods Clin Dev. 2021 Mar 3;21:144-160. doi: 10.1016/j.omtm.2021.02.024. eCollection 2021 Jun 11. Mol Ther Methods Clin Dev. 2021. PMID: 33850950 Free PMC article.