Genome Editing Tools for Lysosomal Storage Disorders
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Publisher
Springer International Publishing
Link
https://link.springer.com/content/pdf/10.1007/978-3-031-33325-5_8
Reference94 articles.
1. Aguilar-González A, González-Correa JE, Barriocanal-Casado E et al (2022) Isogenic GAA-KO murine muscle cell lines mimicking severe Pompe mutations as preclinical models for the screening of potential gene therapy strategies. Int J Mol Sci 23:6298. https://doi.org/10.3390/ijms23116298
2. Allende ML, Cook EK, Larman BC, Nugent A, Brady JM, Golebiowski D, Sena-Esteves M, Tifft CJ, Proia RL (2018) Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation. J Lipid Res 59:550. https://doi.org/10.1194/jlr.M081323
3. Alroy J, Lyons JA (2014) Lysosomal storage diseases. J Inborn Errors Metab Screen 2:2326409813517663. https://doi.org/10.1177/2326409813517663
4. Anzalone AV, Randolph PB, Davis JR et al (2019) Search-and-replace genome editing without double-strand breaks or donor DNA. Nature 576:149. https://doi.org/10.1038/s41586-019-1711-4
5. Anzalone AV, Koblan LW, Liu DR (2020) Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors. Nat Biotechnol 38:824–844
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