scholarly journals Gene repair tool for stem cells

Nature ◽  
2011 ◽  
Vol 474 (7349) ◽  
pp. 8-8
Keyword(s):  
Stem Cells ◽  
Gene Repair

CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease

2017 ◽  
Vol 9 (372) ◽  
pp. eaah3480 ◽  
Author(s):  
Suk See De Ravin ◽  
Linhong Li ◽  
Xiaolin Wu ◽  
Uimook Choi ◽  
Cornell Allen ◽  
...  
Keyword(s):  
Stem Cells ◽  
Hematopoietic Stem Cells ◽  
Chronic Granulomatous Disease ◽  
Granulomatous Disease ◽  
Gene Repair ◽  
Hematopoietic Stem

Towards the Generation of Patient-Specific Pluripotent Stem Cells for Combined Gene and Cell Therapy of Hematologic Disorders

Hematology ◽  
2007 ◽  
Vol 2007 (1) ◽  
pp. 17-22 ◽  
Author(s):  
George Q. Daley
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Cell Therapy ◽  
Somatic Cell ◽  
Pluripotent Stem Cells ◽  
Tissue Transplantation ◽  
Model Systems ◽  
Patient Specific ◽  
Gene Repair ◽  
Hematopoietic Stem

Abstract Hematopoietic stem cell transplantation (HSCT) has proven successful for the treatment of a host of genetic and malignant diseases of the blood, but immune barriers to allogeneic tissue transplantation have hindered wider application. Likewise, gene therapy now appears effective in the treatment of various forms of immune deficiency, and yet insertional mutagenesis from viral gene transfer has raised safety concerns. One strategy for addressing the limitations of both gene therapy and allogeneic transplantation entails the creation of pluripotent stem cells from a patient’s own somatic cells, thereby enabling precise in situ gene repair via homologous recombination in cultured cells, followed by autologous tissue transplantation. In murine model systems, the methods of somatic cell nuclear transfer, parthenogenesis, and direct somatic cell reprogramming with defined genetic factors have been used to generate pluripotent stem cells, and initial efforts at therapeutic gene repair and tissue transplantation suggest that the technology is feasible. Generating patient-specific autologous pluripotent stem cells provides an opportunity to combine gene therapy with autologous cell therapy to treat a host of human conditions. However, a number of technical hurdles must be overcome before therapies based on pluripotent human stem cells will appear in the clinic.

scholarly journals 299. Efficient Functional Oxidase Correction after CRISPR-Oligomer Gene Repair in X-CGD Patient Hematopoietic Stem Cells (HSC) Using Non-Viral, cGMP Compliant, Scalable, Closed System

2015 ◽  
Vol 23 ◽  
pp. S121
Author(s):  
Linhong Li ◽  
Suk see De Ravin ◽  
Cornell Allen ◽  
Pachai Natarajan ◽  
Harry L. Malech ◽  
...  
Keyword(s):  
Stem Cells ◽  
Hematopoietic Stem Cells ◽  
Closed System ◽  
Gene Repair ◽  
Hematopoietic Stem

scholarly journals High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia

2020 ◽  
Vol 26 (2) ◽  
pp. 161-171.e4 ◽  
Author(s):  
Sriram Vaidyanathan ◽  
Ameen A. Salahudeen ◽  
Zachary M. Sellers ◽  
Dawn T. Bravo ◽  
Shannon S. Choi ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Stem Cells ◽  
High Efficiency ◽  
Gene Repair ◽  
Free Gene

scholarly journals Enrichment of G2/M cell cycle phase in human pluripotent stem cells enhances HDR-mediated gene repair with customizable endonucleases

2016 ◽  
Vol 6 (1) ◽  
Author(s):  
Diane Yang ◽  
Marissa A Scavuzzo ◽  
Jolanta Chmielowiec ◽  
Robert Sharp ◽  
Aleksandar Bajic ◽  
...  
Keyword(s):  
Cell Cycle ◽  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
Human Pluripotent Stem Cells ◽  
Cell Cycle Phase ◽  
Cycle Phase ◽  
Gene Repair ◽  
M Cell

scholarly journals Targeted, homology-driven gene insertion in stem cells by ZFN-loaded ‘all-in-one’ lentiviral vectors

eLife ◽  
2016 ◽  
Vol 5 ◽  
Author(s):  
Yujia Cai ◽  
Anders Laustsen ◽  
Yan Zhou ◽  
Chenglong Sun ◽  
Mads Valdemar Anderson ◽  
...  
Keyword(s):  
Stem Cells ◽  
Lentiviral Vector ◽  
Lentiviral Vectors ◽  
Human Stem Cells ◽  
Gene Repair ◽  
Gene Correction ◽  
Gene Therapies ◽  
Gene Insertion ◽  
Insertion Mechanism ◽  
Induced Pluripotent

Biased integration remains a key challenge for gene therapy based on lentiviral vector technologies. Engineering of next-generation lentiviral vectors targeting safe genomic harbors for insertion is therefore of high relevance. In a previous paper (<xref ref-type="bibr" rid="bib2">Cai et al., 2014a</xref>), we showed the use of integrase-defective lentiviral vectors (IDLVs) as carriers of complete gene repair kits consisting of zinc-finger nuclease (ZFN) proteins and repair sequences, allowing gene correction by homologous recombination (HR). Here, we follow this strategy to engineer ZFN-loaded IDLVs that insert transgenes by a homology-driven mechanism into safe loci. This insertion mechanism is driven by time-restricted exposure of treated cells to ZFNs. We show targeted gene integration in human stem cells, including CD34+ hematopoietic progenitors and induced pluripotent stem cells (iPSCs). Notably, targeted insertions are identified in 89% of transduced iPSCs. Our findings demonstrate the applicability of nuclease-loaded ‘all-in-one’ IDLVs for site-directed gene insertion in stem cell-based gene therapies.

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