Targeted genome editing in human long-term repopulating hematopoietic stem cells for the correction of SCID-X1

被引:0
|
作者
Genovese, P. [1 ]
Schiroli, G. [1 ,2 ]
Conway, A. [3 ]
Guschin, D. [3 ]
Escobar, G. [1 ,2 ]
Di Tomaso, T. [1 ]
Marin, V. [4 ]
Giuliani, E. [4 ]
Firrito, C. [1 ]
Gregory, P. D. [3 ]
Holmes, M. C. [3 ]
Gentner, B. [1 ]
Bovolenta, C. [4 ]
Cost, G. J. [3 ]
Lombardo, A. [1 ,2 ]
Naldini, L. [1 ,2 ]
机构
[1] San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy
[2] Univ Vita Salute San Raffaele, Milan, Italy
[3] Sangamo BioSci Inc, Richmond, CA USA
[4] MolMed SpA, Milan, Italy
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
OR012
引用
收藏
页码:A5 / A6
页数:2
相关论文
共 50 条
  • [1] Targeted Genome Editing in Human Long-Term Repopulating Hematopoietic Stem Cells for Correction of SCID-X1
    Genovese, Pietro
    Schiroli, Giulia
    Escobar, Giulia
    Di Tomaso, Tiziano
    Firrito, Claudia
    Calabria, Andrea
    Mazzieri, Roberta
    Gregory, Philip D.
    Holmes, Michael C.
    van der Burg, Mirjam
    Montini, Eugenio
    Gentner, Bernhard
    Lombardo, Angelo
    Naldini, Luigi
    HUMAN GENE THERAPY, 2014, 25 (11) : A31 - A31
  • [2] Site-Specific Genome Editing in Human Long-Term Repopulating Hematopoietic Stem Cells for Correction of SCID-X1
    Genovese, Pietro
    Schiroli, Giulia
    Escobar, Giulia
    Di Tomaso, Tiziano
    Firrito, Claudia
    Moi, Davide
    Benedicenti, Fabrizio
    Calabria, Andrea
    Mazzieri, Roberta
    Gregory, Philip D.
    Holmes, Michael C.
    van der Burg, Mirjam
    Montini, Eugenio
    Gentner, Bernhard
    Lombardo, Angelo
    Naldini, Luigi
    MOLECULAR THERAPY, 2014, 22 : S291 - S291
  • [3] Gene correction for SCID-X1 in long-term hematopoietic stem cells
    Pavel-Dinu, Mara
    Wiebking, Volker
    Dejene, Beruh T.
    Srifa, Waracharee
    Mantri, Sruthi
    Nicolas, Carmencita E.
    Lee, Ciaran
    Bao, Gang
    Kildebeck, Eric J.
    Punjya, Niraj
    Sindhu, Camille
    Inlay, Matthew A.
    Saxena, Nivedita
    DeRavin, Suk See
    Malech, Harry
    Roncarolo, Maria Grazia
    Weinberg, Kenneth, I
    Porteus, Matthew H.
    NATURE COMMUNICATIONS, 2019, 10 (1)
  • [4] Gene correction for SCID-X1 in long-term hematopoietic stem cells
    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
    Nature Communications, 10
  • [5] Correction of SCID-X1 by Targeted Genome Editing of Hematopoietic Stem/Progenitor Cells (HSPC) in the Mouse Model
    Schiroli, Giulia
    Genovese, Pietro
    Capo, Valentina
    Castiello, Maria C.
    Albano, Luisa
    Holmes, Michael C.
    Villa, Anna
    Sitia, Giovanni
    Lombardo, Angelo
    Naldini, Luigi
    MOLECULAR THERAPY, 2016, 24 : S18 - S19
  • [6] Correction of SCID-X1 by targeted genome editing of hematopoietic stem/progenitor cells (HSPC) in the mouse model
    Schiroli, G.
    Genovese, P.
    Capo, V.
    Castiello, M. C.
    Albano, L.
    Sanvito, F.
    Holmes, M. C.
    Villa, A.
    Sitia, G.
    Lombardo, A.
    Naldini, L.
    HUMAN GENE THERAPY, 2016, 27 (11) : A135 - A136
  • [7] Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells
    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
    Nature Communications, 10
  • [8] Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells
    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
    Nature Communications, 10
  • [9] Targeted genome editing in mouse Hematopoietic Stem/Progenitor Cells (HSPC) to model gene correction of SCID-X1
    Schiroli, G.
    Genovese, P.
    Capo, V.
    Castiello, M. C.
    Gregory, P. D.
    Holmes, M. C.
    Sitia, G.
    Villa, A.
    Lombardo, A.
    Naldini, L.
    HUMAN GENE THERAPY, 2015, 26 (10) : A8 - A8
  • [10] Targeted Genome Editing in Mouse Hematopoietic Stem/Progenitor Cells (HSPC) To Model Gene Correction of SCID-X1
    Schiroli, Giulia
    Genovese, Pietro
    Castiello, Maria C.
    Capo, Valentina
    Gregory, Philip D.
    Holmes, Michael C.
    Sitia, Giovanni
    Villa, Anna
    Lombardo, Angelo
    Naldini, Luigi
    MOLECULAR THERAPY, 2015, 23 : S191 - S191