AAV-based in vivo gene therapy for neurological disorders
Author:
Publisher
Springer Science and Business Media LLC
Subject
Drug Discovery,Pharmacology,General Medicine
Link
https://www.nature.com/articles/s41573-023-00766-7.pdf
Reference188 articles.
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2. Leone, P. et al. Long-term follow-up after gene therapy for Canavan disease. Sci. Transl. Med. 4, 165ra163 (2012).
3. Cavazzana, M., Bushman, F. D., Miccio, A., André-Schmutz, I. & Six, E. Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges. Nat. Rev. Drug. Discov. 18, 447–462 (2019).
4. Kinsella, J. L. et al. Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: trial in progress-NCT04201405. Blood 136, 15–16 (2020).
5. Gentner, B. et al. Hematopoietic stem- and progenitor-cell gene therapy for Hurler syndrome. N. Engl. J. Med. 385, 1929–1940 (2021).
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