A severe inherited neuromuscular disease is corrected in mice by intravenous gene delivery.
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06 October 2022
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References
Foust, K.D. et al. Nat. Biotechnol. 28, 271–274 (2010).
Crawford, T. in Neuromuscular Disorders of Infancy, Childhood, and Adolescence: A Clinician's Approach (Jones R.H., De Vivo, D.C. & Darras B.T., eds.) 145–166 (Butterworth Heinemann, Philadelphia, 2003).
Narver, H.L. et al. Ann. Neurol. 64, 465–470 (2008).
Burghes, A.H. & Beattie, C.E. Nat. Rev. Neurosci. 10, 597–609 (2009).
Gavrilina, T.O. et al. Hum. Mol. Genet. 17, 1063–1075 (2008).
Foust, K.D. et al. Nat. Biotechnol. 27, 59–65 (2009).
McCarty, D.M. Mol. Ther. 16, 1648–1656 (2008).
Smith, R.H., Levy, J.R. & Kotin, R.M. Mol. Ther. 17, 1888–1896 (2009).
Clement, N., Knop, D.R. & Byrne, B.J. Hum. Gene Ther. 20, 796–806 (2009).
Finn, J.D. et al. Mol. Ther. 18, 135–142 (2009).
Kao, H.Y. et al. Clin. Chem. 52, 361–369 (2006).
Pyatt, R.E., Mihal, D.C. & Prior, T.W. Clin. Chem. 53, 1879–1885 (2007).
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MacKenzie, A. Genetic therapy for spinal muscular atrophy. Nat Biotechnol 28, 235–237 (2010). https://doi.org/10.1038/nbt0310-235
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DOI: https://doi.org/10.1038/nbt0310-235
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