Sustained Knockdown of a Disease-Causing Gene in Patient-Specific Induced Pluripotent Stem Cells Using Lentiviral Vector-Based Gene Therapy
出版年份 2013 全文链接
标题
Sustained Knockdown of a Disease-Causing Gene in Patient-Specific Induced Pluripotent Stem Cells Using Lentiviral Vector-Based Gene Therapy
作者
关键词
-
出版物
Stem Cells Translational Medicine
Volume 2, Issue 9, Pages 641-654
出版商
Wiley
发表日期
2013-08-08
DOI
10.5966/sctm.2013-0017
参考文献
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。- 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
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExplorePublish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn More