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 50 条

[41] Adeno-associated virus 2-mediated transduction of murine repopulating hematopoietic stem cells and long-term expression of a human globin gene in vivo. [J].

Ponnazhagan, S ;

Yoder, MC ;

Srivastava, A .

BLOOD, 1996, 88 (10) :523-523

[42] 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. [J].

S Cai ;

A Ernstberger ;

S Goebel ;

H Hanenberg ;

K E Pollok .

Pediatric Research, 2006, 60 :496-496

[43] Gene transfer in hematopoietic stem cells with in vivo long-term repopulating ability can be assessed in vitro using the cobblestone area forming cell (CAFC) assay and green fluorescent protein [J].

Zijlmans, JMJM ;

Bierhuizen, MFA ;

Jumelet, E ;

Van Hennik, PB ;

Wognum, AW ;

Wagemaker, G ;

Löwenberg, B ;

Ploemacher, RE .

BRITISH JOURNAL OF HAEMATOLOGY, 1998, 102 (01) :289-289

[44] 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 [J].

Li, Linhong ;

De Ravin, Suk See ;

Allen, Cornell ;

Natarajan, Pachai ;

Malech, Harry L. ;

Peshwa, Madhusudan V. .

MOLECULAR THERAPY, 2015, 23 :S121-S121

[45] Stem Cell Factor-Displaying Simian Immunodeficiency Viral Vectors Together with a Low Conditioning Regimen Allow for Long-Term Engraftment of Gene-Marked Autologous Hematopoietic Stem Cells in Macaques [J].

Verhoeyen, Els ;

Relouzat, Francis ;

Cambot, Marie ;

Costa, Caroline ;

Negre, Didier ;

Legrand, Faezeh ;

Joubert, Christophe ;

Le Grand, Roger ;

Cosset, Francois-Loic ;

Leboulch, Philippe ;

Dubart-Kupperschmitt, Anne ;

Prost, Stephane .

HUMAN GENE THERAPY, 2012, 23 (07) :754-768

[46] Long-Term Effects of Hematopoietic Stem Cell Gene Therapy in the Murine Model of Wiskott-Aldrich Syndrome: Persistence of Functional Correction of T Cells and Lack of Malignant Transformation [J].

Marangoni, Francesco ;

Dupre, Loic ;

Scaramuzza, Samantha ;

Panaroni, Cristina ;

Trifari, Sara ;

Hernandez, Raisa Jofra ;

Thrasher, Adrian J. ;

Galy, Anne ;

Aiuti, Alessandro ;

Naldini, Luigi ;

Roncarolo, Maria Grazia .

MOLECULAR THERAPY, 2006, 13 :S27-S28

[47] Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction (vol 5, 168er1, 2013) [J].

Gaspar, H. B. ;

Cooray, S. ;

Gilmour, K. C. ;

Parsley, K. L. ;

Zhang, F. ;

Adams, S. ;

Bjorkegren, E. ;

Bayford, J. ;

Brown, L. ;

Davies, E. G. ;

Veys, P. ;

Fairbanks, L. ;

Bordon, V. ;

Petropoulou, T. ;

Kinnon, C. ;

Thrasher, A. J. .

SCIENCE TRANSLATIONAL MEDICINE, 2013, 5 (168)

[48] Efficient Genome Editing of the PKLR Locus in Human Long-Term Hematopoietic Stem Cells Using Specific CRISPR/CAS9 RNP and AAV6-Delivery of Donor Templates to Treat Pyruvate Kinase Deficiency [J].

Fananas-Baquero, Sara ;

Quintana-Bustamante, Oscar ;

Alberquilla, Omaira ;

Sanchez-Dominguez, Rebeca ;

Dever, Daniel P. ;

Camarena, Joab ;

Bueren, Juan ;

Porteus, Matthew ;

Segovia, Jose C. .

MOLECULAR THERAPY, 2019, 27 (04) :451-451

[49] Stimulation of mouse bone marrow cells with Kit ligand, FLT3 ligand, and thrombopoietin leads to efficient retrovirus-mediated gene transfer to stem cells, whereas interleukin 3 and interleukin 11 reduce transduction of short- and long-term repopulating cells [J].

Wognum, AW ;

Visser, TP ;

Peters, K ;

Bierhuizen, MFA ;

Wagemaker, G .

HUMAN GENE THERAPY, 2000, 11 (15) :2129-2141

[50] Long Term Follow up after Adoptive Transfer of CD19-Specific CAR+ T Cells Genetically Modified Via Non-Viral Sleeping Beauty S ystem Following Hematopoietic Stem Cell Transplantation (HSCT) [J].

Kebriaei, Partow ;

Huls, Helen ;

Olivares, Simon ;

Orozco, Aaron F. ;

Su, Shihuang ;

Maiti, Sourindra N. ;

De Groot, Elenanor ;

Smith, Amy ;

Yang, Yijun ;

Rondon, Gabriela ;

Qazilbash, Muzaffar H. ;

Ciurea, Stefan O. ;

Alousi, Amin M. ;

Nieto, Yago ;

Marin, David Costa ;

Popat, Uday ;

Hosing, Chitra ;

Kantarjian, Hagop M. ;

Rezvani, Katayoun ;

Shpall, Elizabeth J. ;

Champlin, Richard E. ;

Cooper, Laurence J. N. ;

Singh, Harjeet .

BLOOD, 2017, 130

← 1 2 3 4 5 →