相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。Adeno-associated virus serotype 2 capsid variants for improved liver-directed gene therapy
Nadja Meumann et al.
HEPATOLOGY (2023)
Robust, Durable Gene Activation In Vivo via mRNA-Encoded Activators
Jared P. Beyersdorf et al.
ACS NANO (2022)
AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression
Adrian Westhaus et al.
HUMAN GENE THERAPY (2022)
Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8
Marti Cabanes-Creus et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2022)
CRISPR technologies for precise epigenome editing
Muneaki Nakamura et al.
NATURE CELL BIOLOGY (2021)
Tissue-specific activation of gene expression by the Synergistic Activation Mediator (SAM) CRISPRa system in mice
Charleen Hunt et al.
NATURE COMMUNICATIONS (2021)
High-Throughput In Vitro, Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction
Adrian Westhaus et al.
HUMAN GENE THERAPY (2020)
Artificial escape from XCI by DNA methylation editing of the CDKL5 gene
Julian A. N. M. Halmai et al.
NUCLEIC ACIDS RESEARCH (2020)
X-linked diseases: susceptible females
Barbara R. Migeon
GENETICS IN MEDICINE (2020)
Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2
Marti Cabanes-Creus et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
Restoring the natural tropism of AAV2 vectors for human liver
Marti Cabanes-Creus et al.
SCIENCE TRANSLATIONAL MEDICINE (2020)
Development of AAV Variants with Human Hepatocyte Tropism and Neutralizing Antibody Escape Capacity
Xiaolei Pei et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes
Samantha L. Ginn et al.
JHEP REPORTS (2020)
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality
Marti Cabanes-Creus et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2019)
Superior human hepatocyte transduction with adeno-associated virus vector serotype 7
Wenwei Shao et al.
GENE THERAPY (2019)
Editing the Epigenome: Reshaping the Genomic Landscape
Liad Holtzman et al.
ANNUAL REVIEW OF GENOMICS AND HUMAN GENETICS, VOL 19 (2018)
A mixed modality approach towards Xi reactivation for Rett syndrome and other X-linked disorders
Lieselot L. G. Carrette et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2018)
Rescue of Fragile X Syndrome Neurons by DNA Methylation Editing of the FMR1 Gene
X. Shawn Liu et al.
CELL (2018)
Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity
Nicole K. Paulk et al.
MOLECULAR THERAPY (2018)
In vivo epigenome editing and transcriptional modulation using CRISPR technology
Cia-Hin Lau et al.
TRANSGENIC RESEARCH (2018)
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
Leszek Lisowski et al.
NATURE (2014)
Fiji: an open-source platform for biological-image analysis
Johannes Schindelin et al.
NATURE METHODS (2012)
Epigenetic modifications and human disease
Anna Portela et al.
NATURE BIOTECHNOLOGY (2010)
Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/- mice
Hisaya Azuma et al.
NATURE BIOTECHNOLOGY (2007)
分享论文
