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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK. Mendell JR, et al. Among authors: kaspar bk, kaspar a. N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198. N Engl J Med. 2017. PMID: 29091557 Free article. Clinical Trial.
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.
Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM, Le TT, Morales PR, Rich MM, Burghes AH, Kaspar BK. Foust KD, et al. Among authors: kaspar bk. Nat Biotechnol. 2010 Mar;28(3):271-4. doi: 10.1038/nbt.1610. Epub 2010 Feb 28. Nat Biotechnol. 2010. Retraction in: Nat Biotechnol. 2022 Nov;40(11):1692. doi: 10.1038/s41587-022-01497-7 PMID: 20190738 Free PMC article. Retracted.
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.
Meyer K, Ferraiuolo L, Schmelzer L, Braun L, McGovern V, Likhite S, Michels O, Govoni A, Fitzgerald J, Morales P, Foust KD, Mendell JR, Burghes AH, Kaspar BK. Meyer K, et al. Among authors: kaspar bk. Mol Ther. 2015 Mar;23(3):477-87. doi: 10.1038/mt.2014.210. Epub 2014 Oct 31. Mol Ther. 2015. PMID: 25358252 Free PMC article.
Could gene therapy be the future for muscular dystrophy?
Haidet AM, Mendell JR, Kaspar BK. Haidet AM, et al. Among authors: kaspar bk. Therapy. 2010 May 1;7(3):287-290. doi: 10.2217/thy.10.14. Therapy. 2010. PMID: 20543898 Free PMC article. No abstract available.
116 results