Cargando…

Targeted gene correction of α(1)-antitrypsin deficiency in induced pluripotent stem cells

Human induced pluripotent stem cells (hIPSCs) represent a unique opportunity for regenerative medicine since they offer the prospect of generating unlimited quantities of cells for autologous transplantation as a novel treatment for a broad range of disorders(1,2,3,4). However, the use of hIPSCs in...

Descripción completa

Detalles Bibliográficos
Autores principales:	Yusa, Kosuke, Rashid, S. Tamir, Strick-Marchand, Helene, Varela, Ignacio, Liu, Pei-Qi, Paschon, David E., Miranda, Elena, Ordóñez, Adriana, Hannan, Nick, Rouhani, Foad Jafari, Darche, Sylvie, Alexander, Graeme, Marciniak, Stefan J., Fusaki, Noemi, Hasegawa, Mamoru, Holmes, Michael C., Di Santo, James P., Lomas, David A., Bradley, Allan, Vallier, Ludovic
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2011
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3198846/ https://www.ncbi.nlm.nih.gov/pubmed/21993621 http://dx.doi.org/10.1038/nature10424

Ejemplares similares

hiPSC hepatocyte model demonstrates the role of unfolded protein response and inflammatory networks in α(1)-antitrypsin deficiency
por: Segeritz, Charis-Patricia, et al.
Publicado: (2018)

Stem cell-based therapy for α(1)-antitrypsin deficiency
por: Rashid, S Tamir, et al.
Publicado: (2012)

Mutational History of a Human Cell Lineage from Somatic to Induced Pluripotent Stem Cells
por: Rouhani, Foad J., et al.
Publicado: (2016)

Characterising the association of latency with α(1)-antitrypsin polymerisation using a novel monoclonal antibody
por: Tan, Lu, et al.
Publicado: (2015)

Cargo receptor-assisted endoplasmic reticulum export of pathogenic α1-antitrypsin polymers
por: Ordóñez, Adriana, et al.
Publicado: (2021)

Alpha-1-Antitrypsin Deficiency, the Serpinopathies and Conformational Disease
por: Parmar, Jas S, et al.
Publicado: (2000)

Increased ERK signalling promotes inflammatory signalling in primary airway epithelial cells expressing Z α(1)-antitrypsin
por: van ‘t Wout, Emily F.A., et al.
Publicado: (2014)

A single-chain variable fragment intrabody prevents intracellular polymerization of Z α(1)-antitrypsin while allowing its antiproteinase activity
por: Ordóñez, Adriana, et al.
Publicado: (2015)

The endoplasmic reticulum remains functionally connected by vesicular transport after its fragmentation in cells expressing Z-α(1)-antitrypsin
por: Dickens, Jennifer A., et al.
Publicado: (2016)

Genetic Background Drives Transcriptional Variation in Human Induced Pluripotent Stem Cells
por: Rouhani, Foad, et al.
Publicado: (2014)

The structural basis for Z α(1)-antitrypsin polymerization in the liver
por: Faull, Sarah V., et al.
Publicado: (2020)

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
por: Fusaki, Noemi, et al.
Publicado: (2009)

Reactive centre loop mutants of α-1-antitrypsin reveal position-specific effects on intermediate formation along the polymerization pathway
por: Haq, Imran, et al.
Publicado: (2013)

Reactive centre loop mutants of α-1-antitrypsin reveal position-specific effects on intermediate formation along the polymerization pathway
por: Haq, Imran, et al.
Publicado: (2013)

Non-invasive testing for liver pathology in alpha-1 antitrypsin deficiency
por: Abbas, Syed Hamza, et al.
Publicado: (2020)

Calcium signalling in mammalian cell lines expressing wild type and mutant human α1-Antitrypsin
por: Malintan, Nancy T., et al.
Publicado: (2019)

Emergence of a Stage-Dependent Human Liver Disease Signature with Directed Differentiation of Alpha-1 Antitrypsin-Deficient iPS Cells
por: Wilson, Andrew A., et al.
Publicado: (2015)

Is PiSS Alpha-1 Antitrypsin Deficiency Associated with Disease?
por: McGee, Dawn, et al.
Publicado: (2010)

Altered native stability is the dominant basis for susceptibility of α(1)-antitrypsin mutants to polymerization
por: Irving, James A., et al.
Publicado: (2014)

High-resolution ex vivo NMR spectroscopy of human Z α(1)-antitrypsin
por: Jagger, Alistair M., et al.
Publicado: (2020)

Intrahepatic heteropolymerization of M and Z alpha-1-antitrypsin
por: Laffranchi, Mattia, et al.
Publicado: (2020)

Why has it been so difficult to prove the efficacy of alpha-1-antitrypsin replacement therapy? Insights from the study of disease pathogenesis
por: Dickens, Jennifer A, et al.
Publicado: (2011)

Therapeutic target-site variability in α(1)-antitrypsin characterized at high resolution
por: Patschull, Anathe O. M., et al.
Publicado: (2011)

Development of a small molecule that corrects misfolding and increases secretion of Z α(1)‐antitrypsin
por: Lomas, David A, et al.
Publicado: (2021)

The molecular species responsible for α(1)‐antitrypsin deficiency are suppressed by a small molecule chaperone
por: Ronzoni, Riccardo, et al.
Publicado: (2020)

A Novel Mouse Model for Stable Engraftment of a Human Immune System and Human Hepatocytes
por: Strick-Marchand, Helene, et al.
Publicado: (2015)

Characterisation of a type II functionally-deficient variant of alpha-1-antitrypsin discovered in the general population
por: Laffranchi, Mattia, et al.
Publicado: (2019)

Longer Telomere Length in COPD Patients with α(1)-Antitrypsin Deficiency Independent of Lung Function
por: Saferali, Aabida, et al.
Publicado: (2014)

Alpha-1-antitrypsin augmentation therapy in deficient individuals enrolled in the Alpha-1 Foundation DNA and Tissue Bank
por: Tonelli, Adriano R, et al.
Publicado: (2009)

The role of bronchial epithelial cells in the pathogenesis of COPD in Z-alpha-1 antitrypsin deficiency
por: Pini, Laura, et al.
Publicado: (2014)

The Importance of N186 in the Alpha-1-Antitrypsin Shutter Region Is Revealed by the Novel Bologna Deficiency Variant
por: Ronzoni, Riccardo, et al.
Publicado: (2021)

The pathological Trento variant of alpha‐1‐antitrypsin (E75V) shows nonclassical behaviour during polymerization
por: Miranda, Elena, et al.
Publicado: (2017)

SVIP regulates Z variant alpha-1 antitrypsin retro-translocation by inhibiting ubiquitin ligase gp78
por: Khodayari, Nazli, et al.
Publicado: (2017)

Viral Load Affects the Immune Response to HBV in Mice With Humanized Immune System and Liver
por: Dusséaux, Mathilde, et al.
Publicado: (2017)

Mineralization of alpha-1-antitrypsin inclusion bodies in Mmalton alpha-1-antitrypsin deficiency
por: Callea, Francesco, et al.
Publicado: (2018)

The effects of weekly augmentation therapy in patients with PiZZ α1-antitrypsin deficiency
por: Schmid, ST, et al.
Publicado: (2012)

Crystallographic and Cellular Characterisation of Two Mechanisms Stabilising the Native Fold of α(1)-Antitrypsin: Implications for Disease and Drug Design
por: Gooptu, Bibek, et al.
Publicado: (2009)

Gene therapy for alpha 1-antitrypsin deficiency with an oxidant-resistant human alpha 1-antitrypsin
por: Sosulski, Meredith L., et al.
Publicado: (2020)

Alpha-1 Antitrypsin as a Therapeutic Agent for Conditions not Associated with Alpha-1 Antitrypsin Deficiency
por: Wanner, Adam
Publicado: (2015)

Evaluating the Root Canal Morphology of Permanent Maxillary First Molars in Iranian Population
por: Rezaeian, Mohsen, et al.
Publicado: (2018)

Cannot write session to /tmp/vufind_sessions/sess_brddpvqfglt5nra089j7t2p0ni