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Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Among authors: al zaidy sa. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.
A Roadmap to Newborn Screening for Duchenne Muscular Dystrophy.
Al-Zaidy SA, Lloyd-Puryear M, Kennedy A, Lopez V, Mendell JR. Al-Zaidy SA, et al. Int J Neonatal Screen. 2017 Jun;3(2):8. doi: 10.3390/ijns3020008. Epub 2017 Apr 7. Int J Neonatal Screen. 2017. PMID: 31588416 Free PMC article.
Clinical Follow-Up for Duchenne Muscular Dystrophy Newborn Screening: A Proposal.
Kwon JM, Abdel-Hamid HZ, Al-Zaidy SA, Mendell JR, Kennedy A, Kinnett K, Cwik VA, Street N, Bolen J, Day JW, Connolly AM. Kwon JM, et al. Among authors: al zaidy sa. Muscle Nerve. 2016 Aug;54(2):186-91. doi: 10.1002/mus.25185. Epub 2016 Jun 13. Muscle Nerve. 2016. PMID: 27170260 Free PMC article.
Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion.
Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Mendell JR, et al. Among authors: al zaidy sa. Hum Gene Ther. 2019 Jul;30(7):794-801. doi: 10.1089/hum.2019.006. Epub 2019 Apr 19. Hum Gene Ther. 2019. PMID: 30838895 Free PMC article.
Validation of high-sensitivity assays to quantitate cerebrospinal fluid and serum β-galactosidase activity in patients with GM1-gangliosidosis.
Quadrini KJ, Vrentas C, Duke C, Wilson C, Hinderer CJ, Weinstein DA, Al-Zaidy SA, Browne SE, Wilson JM, Ni YG. Quadrini KJ, et al. Among authors: al zaidy sa. Mol Ther Methods Clin Dev. 2024 Aug 10;32(3):101318. doi: 10.1016/j.omtm.2024.101318. eCollection 2024 Sep 12. Mol Ther Methods Clin Dev. 2024. PMID: 39282076 Free PMC article.
13 results