8 results on '"Laoharawee, Kanut"'
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2. ZFN-mediated in vivo genome editing of hepatocytes results in phenotypic correction in murine MPS I and MPS II models
3. ZFN-mediated in vivo genome editing results in phenotypic correction in murine MPS I and MPS II models
4. Neurologic improvement in a mouse model of Hunter syndrome (mucopolysaccharidosis type II) by treatment with AAV9 vector after the development of cognitive dysfunction
5. Relative effectiveness of different routes of AAV administration for gene therapy of mucopolysaccharidosis
6. AAV9 mediated correction of iduronate-2-sulfatase deficiency in the central nervous system of mucopolysaccharidosis type II mice
7. ZFN-mediated in vivo genome editing results in supraphysiological levels of human iduronate 2-sulfatase and phenotypic correction in a murine MPS II model
8. ZFN-mediated correction of murine MPS I model by expression of the human IDUA cDNA from the albumin “safe harbor” locus
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