AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice
Author:
Publisher
Springer Science and Business Media LLC
Subject
Multidisciplinary
Link
https://www.nature.com/articles/s41586-022-04533-3.pdf
Reference61 articles.
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3. Mendell, J. R. et al. Current clinical applications of in vivo gene therapy with AAVs. Mol. Ther. 29, 464–488 (2020).
4. Hordeaux, J. et al. Adeno-associated virus-induced dorsal root ganglion pathology. Hum. Gene Ther. 31, 808–818 (2020).
5. Van Alstyne, M. et al. Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit. Nat. Neurosci. 24, 930–940 (2021).
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