AAV gene therapy for Tay-Sachs disease
Author:
Funder
BlueGenes Foundation
Publisher
Springer Science and Business Media LLC
Subject
General Biochemistry, Genetics and Molecular Biology,General Medicine
Link
https://www.nature.com/articles/s41591-021-01664-4.pdf
Reference44 articles.
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2. Mendell, J. R. et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N. Engl. J. Med. 377, 1713–1722 (2017).
3. Muramatsu, S. et al. A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson’s disease. Mol. Ther. 18, 1731–1735 (2010).
4. Hwu, W. L. et al. Gene therapy for aromatic L-amino acid decarboxylase deficiency. Sci. Transl. Med. 4, 134ra161 (2012).
5. Passini, M. A. et al. Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-deficient mice. J. Virol. 77, 7034–7040 (2003).
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