Adenine base editing and prime editing of chemically derived hepatic progenitors rescue genetic liver disease

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Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity

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Genome-wide target specificity of CRISPR RNA-guided adenine base editors

(2019) Daesik Kim et al.   NATURE BIOTECHNOLOGY

Adenine base editing in an adult mouse model of tyrosinaemia

(2019) Chun-Qing Song et al.   Nature Biomedical Engineering

Search-and-replace genome editing without double-strand breaks or donor DNA

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Long-Term Outcomes and Practical Considerations in the Pharmacological Management of Tyrosinemia Type 1

(2019) Willem G. van Ginkel et al.   PEDIATRIC DRUGS

Curative Ex Vivo Hepatocyte-Directed Gene Editing in a Mouse Model of Hereditary Tyrosinemia Type 1

(2018) Caitlin VanLith et al.   HUMAN GENE THERAPY

Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction

(2018) Luke W Koblan et al.   NATURE BIOTECHNOLOGY

The polyploid state restricts hepatocyte proliferation and liver regeneration

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Small molecule-mediated reprogramming of human hepatocytes into bipotent progenitor cells

(2018) Yohan Kim et al.   JOURNAL OF HEPATOLOGY

Engineered CRISPR-Cas9 nuclease with expanded targeting space

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Cas9 immunity creates challenges for CRISPR gene editing therapies

(2018) Julie M. Crudele et al.   Nature Communications

Web-based design and analysis tools for CRISPR base editing

(2018) Gue-Ho Hwang et al.   BMC BIOINFORMATICS

Conversion of hepatoma cells to hepatocyte-like cells by defined hepatocyte nuclear factors

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In Vitro Expansion of Primary Human Hepatocytes with Efficient Liver Repopulation Capacity

(2018) Kun Zhang et al.   Cell Stem Cell

Pharmacological induction of a progenitor state for the efficient expansion of primary human hepatocytes

(2018) Carmen Unzu et al.   HEPATOLOGY

In utero CRISPR-mediated therapeutic editing of metabolic genes

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Diagnosis and treatment of tyrosinemia type I: a US and Canadian consensus group review and recommendations

(2017) Jeffrey M Chinsky et al.   GENETICS IN MEDICINE

Cas-analyzer: an online tool for assessing genome editing results using NGS data

(2016) Jeongbin Park et al.   BIOINFORMATICS

Transplantation of a human iPSC-derived hepatocyte sheet increases survival in mice with acute liver failure

(2016) Yasuhito Nagamoto et al.   JOURNAL OF HEPATOLOGY

Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1

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Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia

(2016) Francis P. Pankowicz et al.   Nature Communications

Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases

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Direct Reprogramming of Human Fibroblasts to Functional and Expandable Hepatocytes

(2014) Pengyu Huang et al.   Cell Stem Cell

An Effective Approach to Prevent Immune Rejection of Human ESC-Derived Allografts

(2014) Zhili Rong et al.   Cell Stem Cell

The degree of liver injury determines the role of p21 in liver regeneration and hepatocarcinogenesis in mice

(2013) Laura Elisa Buitrago-Molina et al.   HEPATOLOGY

Tumorigenicity as a clinical hurdle for pluripotent stem cell therapies

(2013) Andrew S Lee et al.   NATURE MEDICINE

Hepatocyte differentiation of mesenchymal stem cells

(2012) Xu-Bo Wu et al.   Hepatobiliary & Pancreatic Diseases International

Rapid generation of mature hepatocyte-like cells from human induced pluripotent stem cells by an efficient three-step protocol

(2011) Yu-Fan Chen et al.   HEPATOLOGY

The Science and Ethics of Induced Pluripotency: What Will Become of Embryonic Stem Cells?

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Direct conversion of mouse fibroblasts to hepatocyte-like cells by defined factors

(2011) Sayaka Sekiya et al.   NATURE

The ploidy conveyor of mature hepatocytes as a source of genetic variation

(2010) Andrew W. Duncan et al.   NATURE

Tyrosinemia Type 1: Metastatic Hepatoblastoma With a Favorable Outcome

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Efficient generation of hepatocyte-like cells from human induced pluripotent stem cells

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Generation of functional human hepatic endoderm from human induced pluripotent stem cells

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(2009) Kyoko Miura et al.   NATURE BIOTECHNOLOGY

Differentiation and Transplantation of Human Embryonic Stem Cell–Derived Hepatocytes

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