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Genome Editing for Mucopolysaccharidoses
Genome editing holds the promise of one-off and potentially curative therapies for many patients with genetic diseases. This is especially true for patients affected by mucopolysaccharidoses as the disease pathophysiology is amenable to correction using multiple approaches. Ex vivo and in vivo genom...
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| Udgivet i: | Int J Mol Sci |
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| Main Authors: | , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
MDPI
2020
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7014411/ https://ncbi.nlm.nih.gov/pubmed/31941077 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms21020500 |
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