Treating Immunodeficiency through HSC Gene Therapy

被引：78

作者：

Booth, Claire ^{[1
,2
]}

Gaspar, H. Bobby ^{[1
,2
]}

Thrasher, Adrian J. ^{[1
,2
]}

机构：

[1] UCL Inst Child Hlth, Mol & Cellular Immunol Sect, London, England

[2] Great Ormond St Hosp Sick Children, Dept Paediat Immunol, London, England

来源：

TRENDS IN MOLECULAR MEDICINE | 2016年 / 22卷 / 04期

基金：

英国惠康基金;

关键词：

CHRONIC GRANULOMATOUS-DISEASE; LEUKOCYTE ADHESION DEFICIENCY; HEMATOPOIETIC STEM-CELLS; ADENOSINE-DEAMINASE-DEFICIENCY; WISKOTT-ALDRICH SYNDROME; LENTIVIRAL VECTOR; LYMPHOPROLIFERATIVE DISEASE; MURINE MODEL; X-CGD; MICE;

D O I：

10.1016/j.molmed.2016.02.002

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Haematopoietic stem cell (HSC) gene therapy has been successfully employed as a therapeutic option to treat specific inherited immune deficiencies, including severe combined immune deficiencies (SCID) over the past two decades. Initial clinical trials using first-generation gamma-retroviral vectors to transfer corrective DNA demonstrated clinical benefit for patients, but were associated with leukemogenesis in a number of cases. Safer vectors have since been developed, affording comparable efficacy with an improved biosafety profile. These vectors are now in Phase I/II clinical trials for a number of immune disorders with more preclinical studies underway. Targeted gene editing allowing precise DNA correction via platforms such as ZFNs, TALENs and CRISPR/Cas9 may now offer promising strategies to improve the safety and efficacy of gene therapy in the future.

引用

页码：317 / 327

页数：11

共 61 条

[1] Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome [J].

Abina, Salima Hacein-Bey ;

Gaspar, H. Bobby ;

Blondeau, Johanna ;

Caccavelli, Laure ;

Charrier, Sabine ;

Buckland, Karen ;

Picard, Capucine ;

Six, Emmanuelle ;

Himoudi, Nourredine ;

Gilmour, Kimberly ;

McNicol, Anne-Marie ;

Hara, Havinder ;

Xu-Bayford, Jinhua ;

Rivat, Christine ;

Touzot, Fabien ;

Mavilio, Fulvio ;

Lim, Annick ;

Treluyer, Jean-Marc ;

Heritier, Sebastien ;

Lefrere, Francois ;

Magalon, Jeremy ;

Pengue-Koyi, Isabelle ;

Honnet, Geraldine ;

Blanche, Stephane ;

Sherman, Eric A. ;

Male, Frances ;

Berry, Charles ;

Malani, Nirav ;

Bushman, Frederic D. ;

Fischer, Alain ;

Thrasher, Adrian J. ;

Galy, Anne ;

Cavazzana, Marina .

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2015, 313 (15) :1550-1563

[2] Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning [J].

Aiuti, A ;

Slavin, S ;

Aker, M ;

Ficara, F ;

Deola, S ;

Mortellaro, A ;

Morecki, S ;

Andolfi, G ;

Tabucchi, A ;

Carlucci, F ;

Marinello, E ;

Cattaneo, F ;

Vai, S ;

Servida, P ;

Miniero, R ;

Roncarolo, MG ;

Bordignon, C .

SCIENCE, 2002, 296 (5577) :2410-2413

[3] Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency. [J].

Aiuti, Alessandro ;

Cattaneo, Federica ;

Galimberti, Stefania ;

Benninghoff, Ulrike ;

Cassani, Barbara ;

Callegaro, Luciano ;

Scaramuzza, Samantha ;

Andolfi, Grazia ;

Mirolo, Massimiliano ;

Brigida, Immacolata ;

Tabucchi, Antonella ;

Carlucci, Filippo ;

Eibl, Martha ;

Aker, Memet ;

Slavin, Shimon ;

Al-Mousa, Hamoud ;

Al Ghonaium, Abdulaziz ;

Ferster, Alina ;

Duppenthaler, Andrea ;

Notarangelo, Luigi ;

Wintergerst, Uwe ;

Buckley, Rebecca H. ;

Bregni, Marco ;

Marktel, Sarah ;

Valsecchi, Maria Grazia ;

Rossi, Paolo ;

Ciceri, Fabio ;

Miniero, Roberto ;

Bordignon, Claudio ;

Roncarolo, Maria-Grazia .

NEW ENGLAND JOURNAL OF MEDICINE, 2009, 360 (05) :447-458

[4]

Aluti A., 2013, SCIENCE, V341

[5] Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors [J].

Bauer, Thomas R., Jr. ;

Allen, James M. ;

Hai, Mehreen ;

Tuschong, Laura M. ;

Khan, Iram F. ;

Olson, Erik M. ;

Adler, Rima L. ;

Burkholder, Tanya H. ;

Gu, Yu-chen ;

Russell, David W. ;

Hickstein, Dennis D. .

NATURE MEDICINE, 2008, 14 (01) :93-97

[6]

Bauer TR, 2000, CURR OPIN MOL THER, V2, P383

[7] Stable and functional lymphoid reconstitution in artemis-deficient mice following lentiviral artemis gene transfer into hematopoietic stem cells [J].

Benjelloun, Fatine ;

Garrigue, Alexandrine ;

Chappedelaine, Corinne Demerens-de ;

Soulas-Sprauel, Pauline ;

Malassis-Seris, Michele ;

Stockholm, Daniel ;

Hauer, Julia ;

Blondeau, Johanna ;

Riviere, Julie ;

Lim, Annick ;

Le Lorc'h, Marc ;

Romana, Serge ;

Brousse, Nicole ;

Paques, Frederique ;

Galy, Anne ;

Charneau, Pierre ;

Fischer, Alain ;

de Villartay, Jean-Pierre ;

Cavazzana-Calvo, Marina .

MOLECULAR THERAPY, 2008, 16 (08) :1490-1499

[8] Restoration of NET formation by gene therapy in CGD controls aspergillosis [J].

Bianchi, Matteo ;

Hakkim, Abdul ;

Brinkmann, Volker ;

Siler, Ulrich ;

Seger, Reinhard A. ;

Zychlinsky, Arturo ;

Reichenbach, Janine .

BLOOD, 2009, 114 (13) :2619-2622

[9] T-LYMPHOCYTE-DIRECTED GENE-THERAPY FOR ADA(-) SCID - INITIAL TRIAL RESULTS AFTER 4 YEARS [J].

BLAESE, RM ;

CULVER, KW ;

MILLER, AD ;

CARTER, CS ;

FLEISHER, T ;

CLERICI, M ;

SHEARER, G ;

CHANG, L ;

CHIANG, YW ;

TOLSTOSHEV, P ;

GREENBLATT, JJ ;

ROSENBERG, SA ;

KLEIN, H ;

BERGER, M ;

MULLEN, CA ;

RAMSEY, WJ ;

MUUL, L ;

MORGAN, RA ;

ANDERSON, WF .

SCIENCE, 1995, 270 (5235) :475-480

[10] GENE-THERAPY IN PERIPHERAL-BLOOD LYMPHOCYTES AND BONE-MARROW FOR ADA(-) IMMUNODEFICIENT PATIENTS [J].

BORDIGNON, C ;

NOTARANGELO, LD ;

NOBILI, N ;

FERRARI, G ;

CASORATI, G ;

PANINA, P ;

MAZZOLARI, E ;

MAGGIONI, D ;

ROSSI, C ;

SERVIDA, P ;

UGAZIO, AG ;

MAVILIO, F .

SCIENCE, 1995, 270 (5235) :470-475

← 1 2 3 4 5 6 7 →