ATP7B gene therapy of autologous reprogrammed hepatocytes alleviates copper accumulation in a mouse model of Wilson’s disease

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

(2021) Yohan Kim et al.   Cell Stem Cell

Extensively expanded murine induced hepatic stem cells maintain high‐efficient hepatic differentiation potential for repopulation of injured livers

(2020) Bing Yu et al.   LIVER INTERNATIONAL

Neurodegeneration in Hepatic and Neurologic Wilson Disease

(2020) André Viveiros et al.   HEPATOLOGY

Liver Expression of a MiniATP7B Gene Results in Long‐Term Restoration of Copper Homeostasis in a Wilson Disease Model in Mice

(2019) Oihana Murillo et al.   HEPATOLOGY

Long-term functional maintenance of primary human hepatocytes in vitro

(2019) Chengang Xiang et al.   SCIENCE

Clinical use of lentiviral vectors

(2018) Michael C. Milone et al.   LEUKEMIA

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

(2018) Yohan Kim et al.   JOURNAL OF HEPATOLOGY

Wilson disease

(2018) Anna Członkowska et al.   Nature Reviews Disease Primers

Expansion and differentiation of human hepatocyte-derived liver progenitor-like cells and their use for the study of hepatotropic pathogens

(2018) Gong-Bo Fu et al.   CELL RESEARCH

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

(2018) Carmen Unzu et al.   HEPATOLOGY

Distinct Gene Expression and Epigenetic Signatures in Hepatocyte-like Cells Produced by Different Strategies from the Same Donor

(2017) Yimeng Gao et al.   Stem Cell Reports

Cell therapy for liver disease: From liver transplantation to cell factory

(2015) Stuart J. Forbes et al.   JOURNAL OF HEPATOLOGY

Wilson's disease and other neurological copper disorders

(2015) Oliver Bandmann et al.   LANCET NEUROLOGY

Current status of human hepatocyte transplantation and its potential for Wilson's disease

(2014) Celine Filippi et al.   Annals of the New York Academy of Sciences

Cell therapy to remove excess copper in Wilson's disease

(2014) Sanjeev Gupta   Annals of the New York Academy of Sciences

Liver transplantation for Wilson's disease

(2014) Michael L. Schilsky   Annals of the New York Academy of Sciences

Pathological mitochondrial copper overload in livers of Wilson's disease patients and related animal models

(2014) Hans Zischka et al.   Annals of the New York Academy of Sciences

Direct Reprogramming of Human Fibroblasts to Functional and Expandable Hepatocytes

(2014) Pengyu Huang et al.   Cell Stem Cell

Wilson Disease Protein ATP7B Utilizes Lysosomal Exocytosis to Maintain Copper Homeostasis

(2014) Elena V. Polishchuk et al.   DEVELOPMENTAL CELL

Transplantation of ATP7B–Transduced Bone Marrow Mesenchymal Stem Cells Decreases Copper Overload in Rats

(2014) Shenglin Chen et al.   PLoS One

Rescue of ATP7B function in hepatocyte-like cells from Wilson's disease induced pluripotent stem cells using gene therapy or the chaperone drug curcumin

(2011) Shiqiang Zhang et al.   HUMAN MOLECULAR GENETICS

Recent Advances in Lentiviral Vector Development and Applications

(2010) Janka Mátrai et al.   MOLECULAR THERAPY

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

(2010) Andrew W. Duncan et al.   NATURE

Preventing and exploiting the oncogenic potential of integrating gene vectors

(2009) Ute Modlich et al.   JOURNAL OF CLINICAL INVESTIGATION

Differentiation and Transplantation of Human Embryonic Stem Cell–Derived Hepatocytes

(2008) Hesham Basma et al.   GASTROENTEROLOGY

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION

Development of cell therapy strategies to overcome copper toxicity in the LEC rat model of Wilson disease

(2008) Harmeet Malhi et al.   Regenerative Medicine