Sustained Knockdown of a Disease-Causing Gene in Patient-Specific Induced Pluripotent Stem Cells Using Lentiviral Vector-Based Gene Therapy

Improved Hepatic Differentiation Strategies for Human Induced Pluripotent Stem Cells

(2013) M. Sgodda et al.   CURRENT MOLECULAR MEDICINE

Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency

(2013) Nunzia Pastore et al.   EMBO Molecular Medicine

Efficient drug screening and gene correction for treating liver disease using patient-specific stem cells

(2013) Su Mi Choi et al.   HEPATOLOGY

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Lentiviral vectors: basic to translational

(2012) Toshie Sakuma et al.   BIOCHEMICAL JOURNAL

Transplantation and Tracking of Human-Induced Pluripotent Stem Cells in a Pig Model of Myocardial Infarction

(2012) Christian Templin et al.   CIRCULATION

Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles

(2012) Christian Mueller et al.   MOLECULAR THERAPY

Alpha-1-antitrypsin deficiency

(2011) Robert Bals   BEST PRACTICE & RESEARCH IN CLINICAL GASTROENTEROLOGY

Modeling abnormal early development with induced pluripotent stem cells from aneuploid syndromes

(2011) Wen Li et al.   HUMAN MOLECULAR GENETICS

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

Lentiviral Vector Design and Imaging Approaches to Visualize the Early Stages of Cellular Reprogramming

(2011) Eva Warlich et al.   MOLECULAR THERAPY

Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

(2011) Kosuke Yusa et al.   NATURE

Site-specific integration and tailoring of cassette design for sustainable gene transfer

(2011) Angelo Lombardo et al.   NATURE METHODS

Humanized mice with ectopic artificial liver tissues

(2011) A. A. Chen et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Alpha-1 antitrypsin is elevated in exhaled breath condensate and serum in exacerbated COPD patients

(2011) A. Rembert Koczulla et al.   RESPIRATORY MEDICINE

In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases

(2011) Vittorio Sebastiano et al.   STEM CELLS

Generation of Healthy Mice from Gene-Corrected Disease-Specific Induced Pluripotent Stem Cells

(2011) Guangming Wu et al.   PLOS BIOLOGY

Hepatic Differentiation of Murine Disease-Specific Induced Pluripotent Stem Cells Allows Disease ModellingIn Vitro

(2011) Reto Eggenschwiler et al.   Stem Cells International

Highly Efficient Reprogramming to Pluripotency and Directed Differentiation of Human Cells with Synthetic Modified mRNA

(2010) Luigi Warren et al.   Cell Stem Cell

A novel monoclonal antibody to characterize pathogenic polymers in liver disease associated with α1-antitrypsin deficiency

(2010) Elena Miranda et al.   HEPATOLOGY

Generation of Human Induced Pluripotent Stem Cells Bearing an Anti-HIV Transgene by a Lentiviral Vector Carrying an Internal Murine Leukemia Virus Promoter

(2010) Masakazu Kamata et al.   HUMAN GENE THERAPY

Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells

(2010) S. Tamir Rashid et al.   JOURNAL OF CLINICAL INVESTIGATION

Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells

(2010) Eirini P Papapetrou et al.   NATURE BIOTECHNOLOGY

Quantitative analysis of conditional gene inactivation using rationally designed, tetracycline-controlled miRNAs

(2010) Stefan M. Berger et al.   NUCLEIC ACIDS RESEARCH

Transgenic α-1-antitrypsin secreted into the bloodstream from salivary glands is biologically active

(2010) P Perez et al.   ORAL DISEASES

Generation of Human Induced Pluripotent Stem Cells by Direct Delivery of Reprogramming Proteins

(2009) Dohoon Kim et al.   Cell Stem Cell

Gene Targeting of a Disease-Related Gene in Human Induced Pluripotent Stem and Embryonic Stem Cells

(2009) Jizhong Zou et al.   Cell Stem Cell

Murine Embryonic Stem Cell-Derived Hepatic Progenitor Cells Engraft in Recipient Livers with Limited Capacity of Liver Tissue Formation

(2009) Amar Deep Sharma et al.   CELL TRANSPLANTATION

Mechanisms controlling titer and expression of bidirectional lentiviral and gammaretroviral vectors

(2009) T Maetzig et al.   GENE THERAPY

Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells

(2009) Ángel Raya et al.   NATURE

Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome

(2009) Noemi FUSAKI et al.   PROCEEDINGS OF THE JAPAN ACADEMY SERIES B-PHYSICAL AND BIOLOGICAL SCIENCES

Genetic diagnosis by whole exome capture and massively parallel DNA sequencing

(2009) Murim Choi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Loss of p21 Permits Carcinogenesis from Chronically Damaged Liver and Kidney Epithelial Cells despite Unchecked Apoptosis

(2008) Holger Willenbring et al.   CANCER CELL

Pluripotent stem cells induced from adult neural stem cells by reprogramming with two factors

(2008) Jeong Beom Kim et al.   NATURE