A solid start for gene therapy in Tay-Sachs disease

doi:10.1038/s41591-022-01687-5

Comment

. 2022 Feb;28(2):236-237.

doi: 10.1038/s41591-022-01687-5.

A solid start for gene therapy in Tay-Sachs disease

Timothy W Yu^{1

2

3

4}, Olaf Bodamer^{5

6

7}

Affiliations

¹ Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA. timothy.yu@childrens.harvard.edu.
² Broad Institute of MIT and Harvard, Cambridge, MA, USA. timothy.yu@childrens.harvard.edu.
³ Harvard Medical School, Boston, MA, USA. timothy.yu@childrens.harvard.edu.
⁴ Department of Neurology, Boston Children's Hospital, Boston, MA, USA. timothy.yu@childrens.harvard.edu.
⁵ Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
⁶ Broad Institute of MIT and Harvard, Cambridge, MA, USA.
⁷ Harvard Medical School, Boston, MA, USA.

PMID: 35145310
DOI: 10.1038/s41591-022-01687-5

Comment

A solid start for gene therapy in Tay-Sachs disease

Timothy W Yu et al. Nat Med. 2022 Feb.

. 2022 Feb;28(2):236-237.

doi: 10.1038/s41591-022-01687-5.

Authors

Timothy W Yu^{1

2

3

4}, Olaf Bodamer^{5

6

7}

Affiliations

¹ Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA. timothy.yu@childrens.harvard.edu.
² Broad Institute of MIT and Harvard, Cambridge, MA, USA. timothy.yu@childrens.harvard.edu.
³ Harvard Medical School, Boston, MA, USA. timothy.yu@childrens.harvard.edu.
⁴ Department of Neurology, Boston Children's Hospital, Boston, MA, USA. timothy.yu@childrens.harvard.edu.
⁵ Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
⁶ Broad Institute of MIT and Harvard, Cambridge, MA, USA.
⁷ Harvard Medical School, Boston, MA, USA.

PMID: 35145310
DOI: 10.1038/s41591-022-01687-5

No abstract available

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Comment on

AAV gene therapy for Tay-Sachs disease.
Flotte TR, Cataltepe O, Puri A, Batista AR, Moser R, McKenna-Yasek D, Douthwright C, Gernoux G, Blackwood M, Mueller C, Tai PWL, Jiang X, Bateman S, Spanakis SG, Parzych J, Keeler AM, Abayazeed A, Rohatgi S, Gibson L, Finberg R, Barton BA, Vardar Z, Shazeeb MS, Gounis M, Tifft CJ, Eichler FS, Brown RH Jr, Martin DR, Gray-Edwards HL, Sena-Esteves M. Flotte TR, et al. Nat Med. 2022 Feb;28(2):251-259. doi: 10.1038/s41591-021-01664-4. Epub 2022 Feb 10. Nat Med. 2022. PMID: 35145305 Free PMC article.

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References

1. Flotte, T. R. et al. Nat. Med. https://doi.org/10.1038/s41591-021-01664-4 (2022). - DOI - PubMed
1. Platt, F. M., d’Azzo, A., Davidson, B. L., Neufeld, E. F. & Tifft, C. J. Nat. Rev. Dis. Primers 4, 274 (2018).
1. Bembi, B. et al. Neurology 66, 278–280 (2006). - DOI
1. Maegawa, G. H. B. et al. Mol. Genet. Metab. 98, 215–224 (2009). - DOI
1. Hwu, W. L. et al. Sci. Transl. Med. 4, 134ra61 (2012). - DOI

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[1] Flotte, T. R. et al. Nat. Med. https://doi.org/10.1038/s41591-021-01664-4 (2022). - DOI - PubMed

[2] Flotte, T. R. et al. Nat. Med. https://doi.org/10.1038/s41591-021-01664-4 (2022). - DOI - PubMed

[3] Platt, F. M., d’Azzo, A., Davidson, B. L., Neufeld, E. F. & Tifft, C. J. Nat. Rev. Dis. Primers 4, 274 (2018).

[4] Platt, F. M., d’Azzo, A., Davidson, B. L., Neufeld, E. F. & Tifft, C. J. Nat. Rev. Dis. Primers 4, 274 (2018).

[5] Bembi, B. et al. Neurology 66, 278–280 (2006). - DOI

[6] Bembi, B. et al. Neurology 66, 278–280 (2006). - DOI

[7] Maegawa, G. H. B. et al. Mol. Genet. Metab. 98, 215–224 (2009). - DOI

[8] Maegawa, G. H. B. et al. Mol. Genet. Metab. 98, 215–224 (2009). - DOI

[9] Hwu, W. L. et al. Sci. Transl. Med. 4, 134ra61 (2012). - DOI

[10] Hwu, W. L. et al. Sci. Transl. Med. 4, 134ra61 (2012). - DOI

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A solid start for gene therapy in Tay-Sachs disease

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A solid start for gene therapy in Tay-Sachs disease

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