Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes-Reference-Cited by-同舟云学术

Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes

Published:2019-10 Issue:4 Volume:13 Page:590-598
ISSN:2213-6711
Container-title:Stem Cell Reports
language:en
Short-container-title:Stem Cell Reports

Author:

Klatt Denise,Cheng Erica,Philipp Friederike,Selich Anton,Dahlke Julia,Schmidt Reinhold E.,Schott Juliane W.,Büning Hildegard,Hoffmann Dirk,Thrasher Adrian J.,Schambach Axel

Funder

German Research Foundation

REBIRTH Cluster of Excellence

Federal Ministry of Education and Research

iMACnet

German Academic Scholarship Foundation

Horizon 2020

Wellcome Trust

NIHR Biomedical Research Centre

NHS Foundation Trust

University College London

Publisher

Elsevier BV

Subject

Cell Biology,Developmental Biology,Genetics,Biochemistry

Reference21 articles.

1. Bioreactor-based mass production of human iPSC-derived macrophages enables immunotherapies against bacterial airway infections;Ackermann;Nat. Commun.,2018

2. Promoter and lineage independent anti-silencing activity of the A2 ubiquitous chromatin opening element for optimized human pluripotent stem cell-based gene therapy;Ackermann;Biomaterials,2014

3. Allogeneic hematopoietic cell transplantation for chronic granulomatous disease: controversies and state of the art;Connelly;J. Pediatr. Infect. Dis. Soc.,2018

4. NCF1 gene and pseudogene pattern: association with parasitic infection and autoimmunity;Greve;Malar. J.,2008

5. Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study;Güngör;Lancet,2014

Cited by 20 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Targeted knock-in of NCF1 cDNA into the NCF2 locus leads to myeloid phenotypic correction of p47 -deficient chronic granulomatous disease;Molecular Therapy - Nucleic Acids;2024-09

2. Functional neutrophil disorders: Chronic granulomatous disease and beyond;Immunological Reviews;2024-03

3. Complications pulmonaires de la granulomatose septique chronique;Revue des Maladies Respiratoires;2024-02

4. Recent Advances in CRISPR/Cas9 Delivery Approaches for Therapeutic Gene Editing of Stem Cells;Stem Cell Reviews and Reports;2023-09-18

5. A Model of iPSC-Derived Macrophages with TNFAIP3 Overexpression Reveals the Peculiarities of TNFAIP3 Protein Expression and Function in Human Macrophages;International Journal of Molecular Sciences;2023-08-16