Gene correction for SCID-X1 in long-term hematopoietic stem cells

QR Code

Gene correction for SCID-X1 in long-term hematopoietic stem cells

Gene correction in hematopoietic stem cells could be a powerful way to treat monogenic diseases of the blood and immune system. Here the authors develop a strategy using CRISPR-Cas9 and an aAdeno-Associated vVirus(AAV)-delivered IL2RG cDNA to correct X-linked sSevere Ccombined iImmunodeficiency (SCI...

Description complète

Enregistré dans:

Détails bibliographiques
Auteurs principaux:	Mara Pavel-Dinu, Volker Wiebking, Beruh T. Dejene, Waracharee Srifa, Sruthi Mantri, Carmencita E. Nicolas, Ciaran Lee, Gang Bao, Eric J. Kildebeck, Niraj Punjya, Camille Sindhu, Matthew A. Inlay, Nivedita Saxena, Suk See DeRavin, Harry Malech, Maria Grazia Roncarolo, Kenneth I. Weinberg, Matthew H. Porteus
Format:	article
Langue:	EN
Publié:	Nature Portfolio 2019
Sujets:	Science Q
Accès en ligne:	https://doaj.org/article/66cf921ac2ec4178bebe47ca77ff7c87
Tags:	Ajouter un tag Pas de tags, Soyez le premier à ajouter un tag!

Documents similaires

Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells
par: Mara Pavel-Dinu, et autres
Publié: (2019)

Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells
par: Mara Pavel-Dinu, et autres
Publié: (2019)

Cas9-AAV6-engineered human mesenchymal stromal cells improved cutaneous wound healing in diabetic mice
par: Waracharee Srifa, et autres
Publié: (2020)

Reevaluation of NOD/SCID Mice as NK Cell-Deficient Models
par: Miao Miao, et autres
Publié: (2021)

Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy
par: Federico A. Moretti, et autres
Publié: (2021)

Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID
par: Claire Booth, et autres
Publié: (2009)

Identification and Characterization of Novel Rat Polyomavirus 2 in a Colony of X-SCID Rats by P-PIT assay
par: Lora H. Rigatti, et autres
Publié: (2016)

Antiviral activity of Bay 41-4109 on hepatitis B virus in humanized Alb-uPA/SCID mice.
par: Nicolas Brezillon, et autres
Publié: (2011)

CRISPR/Cas9-Mediated Deletion of Foxn1 in NOD/SCID/IL2rg−/− Mice Results in Severe Immunodeficiency
par: Xinru Wei, et autres
Publié: (2017)

Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
par: Natalia Gomez-Ospina, et autres
Publié: (2019)

Molecular imaging of myogenic stem/progenitor cells with [18F]-FHBG PET/CT system in SCID mice model of post-infarction heart
par: Weronika Wargocka-Matuszewska, et autres
Publié: (2021)

Cryptosporidium parvum infection in SCID mice infected with only one oocyst: qPCR assessment of parasite replication in tissues and development of digestive cancer.
par: Sadia Benamrouz, et autres
Publié: (2012)

Improved function and proliferation of adult human beta cells engrafted in diabetic immunodeficient NOD-scid IL2rγnull mice treated with alogliptin
par: Jurczyk A, et autres
Publié: (2013)

Antibody repertoires in humanized NOD-scid-IL2Rγ(null) mice and human B cells reveals human-like diversification and tolerance checkpoints in the mouse.
par: Gregory C Ippolito, et autres
Publié: (2012)

NOD/scid IL‐2Rγnull mice reconstituted with peripheral blood mononuclear cells from patients with Crohn's disease reflect the human pathological phenotype
par: Anna‐Lena Unterweger, et autres
Publié: (2021)

The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells
par: Rajiv Sharma, et autres
Publié: (2021)

Lixisenatide accelerates restoration of normoglycemia and improves human beta-cell function and survival in diabetic immunodeficient NOD–scid IL-2rgnull RIP-DTR mice engrafted with human islets
par: Yang C, et autres
Publié: (2015)

Intratibial injection of human multiple myeloma cells in NOD/SCID IL-2Rγ(null) mice mimics human myeloma and serves as a valuable tool for the development of anticancer strategies.
par: Julia Schueler, et autres
Publié: (2013)

Intraperitoneal administration of human "Neo-Islets", 3-D organoids of mesenchymal stromal and pancreatic islet cells, normalizes blood glucose levels in streptozotocin-diabetic NOD/SCID mice: Significance for clinical trials.
par: Christof Westenfelder, et autres
Publié: (2021)

Intraperitoneal administration of human “Neo-Islets”, 3-D organoids of mesenchymal stromal and pancreatic islet cells, normalizes blood glucose levels in streptozotocin-diabetic NOD/SCID mice: Significance for clinical trials
par: Christof Westenfelder, et autres
Publié: (2021)

Human delta like 1-expressing human mesenchymal stromal cells promote human T cell development and antigen-specific response in humanized NOD/SCID/IL-2R $$\upgamma $$ γ null (NSG) mice
par: Do Hee Kwon, et autres
Publié: (2021)

Functional significance of U2AF1 S34F mutations in lung adenocarcinomas
par: Mohammad S. Esfahani, et autres
Publié: (2019)

Impact of climate change on biodiversity and associated key ecosystem services in Africa: a systematic review
par: Dejene W. Sintayehu
Publié: (2018)

Replacing The Responsibility to Protect: The Equitable Theory of Humanitarian Intervention
par: Ciarán Burke
Publié: (2009)

Ernesto Mayz Vallenilla. In memoriam. (1925-2015). Rememorando una clase magistral. (A modo de homenaje personal)
par: Dinu Garber E.
Publié: (2016)

Enrichment of soil organic carbon by native earthworms in a patch of tropical soil, Kerala, India: First report
par: S. N. Sruthi, et autres
Publié: (2018)

Les pratiques de nomination des migrants irlandais en Espagne (1600-1680) : entre le rejet et l'assimilation
par: Ciaran O' Scea
Publié: (2015)

Corrigendum: Early ERP Evidence for Children's and Adult's Sensitivity to Scalar Implicatures Triggered by Existential Quantifiers (Some)
par: Daniele Panizza, et autres
Publié: (2021)

Early ERP Evidence for Children’s and Adult’s Sensitivity to Scalar Implicatures Triggered by Existential Quantifiers (Some)
par: Daniele Panizza, et autres
Publié: (2021)

KEHIDUPAN WAYANG GOLEK MENAK KEBUMEN PADA MASA KI SINDHU JOTARYONO (1955-1980)
par: Sunarto Sindhu
Publié: (2016)

Utilisation of Supplementary Nutrition Service at Anganwadi Centres in a Block of Ganjam District, Odisha: A Cross-sectional Study
par: Smaranita Sabat, et autres
Publié: (2021)

Performance of polycaprolactone/TiO2 composite membrane for the effective treatment of dairy effluents
par: Sankar Nivedita, et autres
Publié: (2021)

Emergence of a field-driven U(1) spin liquid in the Kitaev honeycomb model
par: Ciarán Hickey, et autres
Publié: (2019)

A simple computational algorithm with inertial extrapolation for generalized split common fixed point problems
par: Anteneh Getachew Gebrie, et autres
Publié: (2021)

Teachers’ misconception concerning valence and valency in chemistry
par: Raghubir Singh Sindhu
Publié: (2021)

Differences in the behaviour of model and non-model species of ants in interactions with the pod-sucking myrmecomorphic bug, Riptortus linearis (Hemiptera: Alydidae)
par: Joy Nivedita SAMUEL, et autres
Publié: (2021)

A Far Cry from School History: Massive Online Open Courses as a Generative Source for Historical Research
par: Silvia Elena Gallagher, et autres
Publié: (2016)

Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing
par: Samantha G. Scharenberg, et autres
Publié: (2020)

Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
par: Adam C. Wilkinson, et autres
Publié: (2021)

Author Correction: Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing
par: Samantha G. Scharenberg, et autres
Publié: (2020)