Search Results - "Liao, Sophia H Y"
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Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation
Published in Nature communications (14-03-2024)“…Developing clinically predictive model systems for evaluating gene transfer and gene editing technologies has become increasingly important in the era of…”
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2
Identification of liver-specific enhancer–promoter activity in the 3′ untranslated region of the wild-type AAV2 genome
Published in Nature genetics (01-08-2017)“…Ian Alexander and colleagues characterize a liver-specific enhancer–promoter element that is found in the genome of wild-type adeno-associated virus type 2…”
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3
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality
Published in Molecular therapy. Methods & clinical development (15-03-2019)“…Adeno-associated virus (AAV) vectors have become one of the most widely used gene transfer tools in human gene therapy. Considerable effort is currently being…”
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4
Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency
Published in Molecular therapy. Nucleic acids (17-03-2017)“…In early gene therapy trials for SCID-X1, using γ-retroviral vectors, T cell leukemias developed in a subset of patients secondary to insertional…”
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5
Development of CNS tropic AAV1-like variants with reduced liver-targeting following systemic administration in mice
Published in Molecular therapy (06-03-2024)“…Directed evolution of natural AAV9 using peptide display libraries have been widely used in the search for an optimal recombinant AAV (rAAV) for transgene…”
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Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8
Published in Molecular therapy. Methods & clinical development (10-03-2022)“…Recent clinical successes have intensified interest in using adeno-associated virus (AAV) vectors for therapeutic gene delivery. The liver is a key clinical…”
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Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2
Published in Molecular therapy. Methods & clinical development (12-06-2020)“…Use of the prototypical adeno-associated virus type 2 (AAV2) capsid delivered unexpectedly modest efficacy in an early liver-targeted gene therapy trial for…”
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Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes
Published in Molecular therapy. Methods & clinical development (14-09-2023)“…Recombinant adeno-associated viruses (rAAVs) have emerged as one of the most promising gene therapy vectors that have been successfully used in pre-clinical…”
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Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids
Published in Molecular therapy. Methods & clinical development (11-06-2021)“…Recent successes in clinical gene therapy applications have intensified the interest in using adeno-associated viruses (AAVs) as vectors for gene delivery into…”
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Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution
Published in Molecular therapy. Methods & clinical development (09-03-2023)“…Recent clinical successes have intensified interest in using adeno-associated virus (AAV) vectors for therapeutic gene delivery. The liver is a key clinical…”
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Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes
Published in JHEP reports (01-02-2020)“…Genome editing technology has immense therapeutic potential and is likely to rapidly supplant contemporary gene addition approaches. Key advantages include the…”
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Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and γc Overexpression
Published in Molecular therapy (01-05-2010)“…The development of leukemia as a consequence of vector-mediated genotoxicity in gene therapy trials for X-linked severe combined immunodeficiency (SCID-X1) has…”
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Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression
Published in Molecular therapy (01-05-2010)“…The development of leukemia as a consequence of vector-mediated genotoxicity in gene therapy trials for X-linked severe combined immunodeficiency (SCID-X1) has…”
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