Non-viral DNA delivery associated to TALEN® gene editing leads to highly efficient correction of sickle cell mutation in long-term repopulating hematopoietic stem cells

被引：0

作者：

Moiani, A. ^{[1
]}

Letort, G. ^{[1
]}

Lizot, S. ^{[1
]}

Hong, P. ^{[1
]}

Temburni-Blake, S. ^{[1
]}

Felix, T. ^{[2
]}

Chalumeau, A. ^{[2
]}

Miccio, A. ^{[2
]}

Gouble, A. ^{[1
]}

Juillerat, A. ^{[1
]}

Duclert, A. ^{[1
]}

Duchateau, P. ^{[1
]}

Valton, J. ^{[1
]}

机构：

[1] Cellectis Therapeut, Paris, France

[2] Imagine Inst, Paris, France

来源：

HUMAN GENE THERAPY | 2022年 / 33卷 / 23-24期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

OR46

引用

页码：A17 / A17

页数：1

共 48 条

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[32] Highly Efficient Gene-Editing of S. aureus Cas9 in Patient Hematopoietic Stem and Progenitor Cells for Treating Sickle Cell Disease
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[33] Non-Viral RNA-Lipid Nanoparticles for High Efficiency Genome Editing of CD34+Hematopoietic Stem and Progenitor Cells for Advanced Cell and Gene Therapies
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[34] Non-Viral RNA-Lipid Nanoparticles for High Efficiency Genome Editing of CD34+Hematopoietic Stem and Progenitor Cells for Advanced Cell and Gene Therapies
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[35] Chemoprotection of long-term repopulating hematopoietic stem cells from alkylator therapy:: In vivo comparison of gene-transfer vectors that express a DNA repair protein
Cai, S.
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[36] Innovative Collagen Nano-Hydroxyapatite Scaffolds Offer a Highly Efficient Non-Viral Gene Delivery Platform for Stem Cell-Mediated Bone Formation
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[37] Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction
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Gilmour, K. C.
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Zhang, F.
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Fairbanks, L.
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[38] Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction
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[39] Adeno-associated virus 2-mediated transduction of murine repopulating hematopoietic stem cells and long-term expression of a human globin gene in vivo.
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[40] 36 Chemoprotection Of Long-Term Repopulating Hematopoietic Stem Cells From Alkylator Therapy: in vivo Comparison of Gene-Transfer Vectors that Express a DNA Repair Protein.
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S Goebel
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Pediatric Research, 2006, 60 : 496 - 496

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