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Sophia H.Y. Liao

Children's Medical Research Institute

10

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Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids

2021 Molecular therapy. Methods & clinical development
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Novel human liver-tropic AAV variants generated by capsid shuffling define transferable domains that markedly enhance the human tropism of AAV7 and AAV8.

2021 Molecular therapy. Methods & clinical development
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Restoring the natural tropism of AAV2 vectors for human liver.

2020 Science Translational Medicine
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Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2.

2020 Molecular therapy. Methods & clinical development
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Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes

2019
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Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality

2019 Molecular therapy. Methods & clinical development
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Identification of liver-specific enhancer-promoter activity in the 3[prime] untranslated region of the wild-type AAV2 genome

2017 Nature Genetics
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Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency

2017 Molecular therapy. Nucleic acids
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Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and γc Overexpression

2010 Molecular Therapy
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Lymphomagenesis in SCID-X1 mice independent of insertional mutagenesis and gamma c over-expression

2009
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