Clinical Translation of TALENS: Treating SCID-X1 by Gene Editing in iPSCs

被引：7

作者：

Biffi, Alessandra ^{[1
]}

机构：

[1] Ist Sci San Raffaele, San Raffaele Telethon Inst Gene, I-20132 Milan, Italy

来源：

CELL STEM CELL | 2015年 / 16卷 / 04期

关键词：

SEVERE COMBINED IMMUNODEFICIENCY; HEMATOPOIETIC STEM-CELLS; THERAPY;

D O I：

10.1016/j.stem.2015.03.009

中图分类号：

Q813 [细胞工程];

学科分类号：

摘要：

Mutations causing X-linked severe combined immunodeficiency (SCID-X1) reduce immune cell populations and function and may be amenable to targeted gene correction strategies. Now in Cell Stem Cell, Menon et al. (2015) correct SCID-X1-related blood differentiation defects by TALEN-mediated genome editing in patient-derived iPSCs, suggesting a possible strategy for autologous cell therapy of SCID-X1.

引用

页码：348 / 349

页数：2

共 9 条

[1] Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement [J].

Aiuti, A ;

Vai, S ;

Mortellaro, A ;

Casorati, G ;

Ficara, F ;

Andolfi, G ;

Ferrari, G ;

Tabucchi, A ;

Carlucci, F ;

Ochs, HD ;

Notarangelo, LD ;

Roncarolo, MG ;

Bordignon, C .

NATURE MEDICINE, 2002, 8 (05) :423-425

[2] Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies:: report of the European experience 1968-99 [J].

Antoine, C ;

Müller, S ;

Cant, A ;

Cavazzana-Calvo, M ;

Veys, P ;

Vossen, J ;

Fasth, A ;

Heilmann, C ;

Wulffraat, N ;

Seger, R ;

Blanche, S ;

Friedrich, W ;

Abinun, M ;

Davies, G ;

Bredius, R ;

Schulz, A ;

Landais, P ;

Fischer, A .

LANCET, 2003, 361 (9357) :553-560

[3] Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening [J].

Brown, Lucinda ;

Xu-Bayford, Jinhua ;

Allwood, Zoe ;

Slatter, Mary ;

Cant, Andrew ;

Davies, E. Graham ;

Veys, Paul ;

Gennery, Andrew R. ;

Gaspar, H. Bobby .

BLOOD, 2011, 117 (11) :3243-3246

[4] Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better? [J].

Gennery, Andrew R. ;

Slatter, Mary A. ;

Grandin, Laure ;

Taupin, Pierre ;

Cant, Andrew J. ;

Veys, Paul ;

Amrolia, Persis J. ;

Gaspar, H. Bobby ;

Davies, E. Graham ;

Friedrich, Wilhelm ;

Hoenig, Manfred ;

Notarangelo, Luigi D. ;

Mazzolari, Evelina ;

Porta, Fulvio ;

Bredius, Robbert G. M. ;

Lankester, Arjen C. ;

Wulffraat, Nico M. ;

Seger, Reinhard ;

Guengoer, Tayfun ;

Fasth, Anders ;

Sedlacek, Petr ;

Neven, Benedicte ;

Blanche, Stephane ;

Fischer, Alain ;

Cavazzana-Calvo, Marina ;

Landais, Paul .

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 2010, 126 (03) :602-U342

[5] Targeted genome editing in human repopulating haematopoietic stem cells [J].

Genovese, Pietro ;

Schiroli, Giulia ;

Escobar, Giulia ;

Di Tomaso, Tiziano ;

Firrito, Claudia ;

Calabria, Andrea ;

Moi, Davide ;

Mazzieri, Roberta ;

Bonini, Chiara ;

Holmes, Michael C. ;

Gregory, Philip D. ;

van der Burg, Mirjam ;

Gentner, Bernhard ;

Montini, Eugenio ;

Lombardo, Angelo ;

Naldini, Luigi .

NATURE, 2014, 510 (7504) :235-+

[6] Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy [J].

Hacein-Bey-Abina, S ;

Le Deist, F ;

Carlier, F ;

Bouneaud, C ;

Hue, C ;

De Villartay, JP ;

Thrasher, AJ ;

Wulffraat, N ;

Sorensen, R ;

Dupuis-Girod, S ;

Fischer, A ;

Cavazzana-Calvo, M ;

Davies, EG ;

Kuis, W ;

Lundlaan, WHK ;

Leiva, L .

NEW ENGLAND JOURNAL OF MEDICINE, 2002, 346 (16) :1185-1193

[7] A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency [J].

Hacein-Bey-Abina, S. ;

Pai, S. -Y. ;

Gaspar, H. B. ;

Armant, M. ;

Berry, C. C. ;

Blanche, S. ;

Bleesing, J. ;

Blondeau, J. ;

de Boer, H. ;

Buckland, K. F. ;

Caccavelli, L. ;

Cros, G. ;

De Oliveira, S. ;

Fernandez, K. S. ;

Guo, D. ;

Harris, C. E. ;

Hopkins, G. ;

Lehmann, L. E. ;

Lim, A. ;

London, W. B. ;

van der Loo, J. C. M. ;

Malani, N. ;

Male, F. ;

Malik, P. ;

Marinovic, M. A. ;

McNicol, A. -M. ;

Moshous, D. ;

Neven, B. ;

Oleastro, M. ;

Picard, C. ;

Ritz, J. ;

Rivat, C. ;

Schambach, A. ;

Shaw, K. L. ;

Sherman, E. A. ;

Silberstein, L. E. ;

Six, E. ;

Touzot, F. ;

Tsytsykova, A. ;

Xu-Bayford, J. ;

Baum, C. ;

Bushman, F. D. ;

Fischer, A. ;

Kohn, D. B. ;

Filipovich, A. H. ;

Notarangelo, L. D. ;

Cavazzana, M. ;

Williams, D. A. ;

Thrasher, A. J. .

NEW ENGLAND JOURNAL OF MEDICINE, 2014, 371 (15) :1407-1417

[8] LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 [J].

Hacein-Bey-Abina, S ;

Von Kalle, C ;

Schmidt, M ;

McCcormack, MP ;

Wulffraat, N ;

Leboulch, P ;

Lim, A ;

Osborne, CS ;

Pawliuk, R ;

Morillon, E ;

Sorensen, R ;

Forster, A ;

Fraser, P ;

Cohen, JI ;

de Saint Basile, G ;

Alexander, I ;

Wintergerst, U ;

Frebourg, T ;

Aurias, A ;

Stoppa-Lyonnet, D ;

Romana, S ;

Radford-Weiss, I ;

Gross, F ;

Valensi, F ;

Delabesse, E ;

Macintyre, E ;

Sigaux, F ;

Soulier, J ;

Leiva, LE ;

Wissler, M ;

Prinz, C ;

Rabbitts, TH ;

Le Deist, F ;

Fischer, A ;

Cavazzana-Calvo, M .

SCIENCE, 2003, 302 (5644) :415-419

[9] Lymphoid Regeneration from Gene-Corrected SCID-X1 Subject-Derived iPSCs [J].

Menon, Tushar ;

Firth, Amy L. ;

Scripture-Adams, Deirdre D. ;

Galic, Zoran ;

Qualls, Susan J. ;

Gilmore, William B. ;

Ke, Eugene ;

Singer, Oded ;

Anderson, Leif S. ;

Bornzin, Alexander R. ;

Alexander, Ian E. ;

Zack, Jerome A. ;

Verma, Inder M. .

CELL STEM CELL, 2015, 16 (04) :367-372

← 1 →