Gene therapy of inherited immunodeficiencies.
作者： Giorgia Santilli , Susannah I Thornhill , Christine Kinnon , Adrian J Thrasher
关键词: Wiskott–Aldrich syndrome 、 Immunology 、 Chronic granulomatous disease 、 Severe combined immunodeficiency 、 Genetic enhancement 、 Clinical trial 、 Transplantation 、 Viral vector 、 Adenosine deaminase deficiency 、 Bioinformatics 、 Biology
摘要: Background: Primary immunodeficiencies (PID) are a group of inherited diseases that affect the development or activity immune system. In severe cases allogeneic haematopoietic stem cell transplantation has proved to be successful curative modality but it is limited by toxicity and reduced efficacy in mismatched donor settings. Objective: Gene therapy for PID been developed as an alternative strategy entered clinical arena. this review we discuss outcomes recent gene trials some problems remain tackled. Methods: Results from X-linked combined immunodeficiency (SCID-X1), adenosine deaminase deficient SCID (ADA-SCID), chronic granulomatous disease (X-CGD) discussed. addition, other conditions highlighted such Wiskott Aldrich Syndrome (WAS) which shown considerable promise preclinical studies, currently being translated into novel approaches. Resul...
informahealthcare.com参考文章(93)
V W van Beusechem, L D Fairbanks, D Valerio, M Debree, A Moseley, R J Levinsky, J L Perignon, B Gaspar, C H Skeoch, A Fischer, P M Hoogerbrugge, F le Deist, M Harvey, G Morgan, Bone marrow gene transfer in three patients with adenosine deaminase deficiency. Gene Therapy. ,vol. 3, pp. 179- 183 ,(1996)
Ricardo A Dewey, Kaan Boztug, Christoph Klein, Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Current Opinion in Molecular Therapeutics. ,vol. 8, pp. 390- 395 ,(2006)
Anders Fasth, Paul Landais, Wilhelm Friedrich, M. Cavazzana-Calvo, Bert Gerritsen, Gareth Morgan, Jaak Vossen, Paul Veys, Susannah Müller, Andrew Cant, Theresa Espanol, Elie Haddad, Alain Fischer, Yves Bertrand, Fulvio Porta, Long-Term Immune Reconstitution and Outcome After HLA-Nonidentical T-Cell–Depleted Bone Marrow Transplantation for Severe Combined Immunodeficiency: A European Retrospective Study of 116 Patients Blood. ,vol. 91, pp. 3646- 3653 ,(1998) , 10.1182/BLOOD.V91.10.3646
Frederic D Bushman, Targeting survival: integration site selection by retroviruses and LTR-retrotransposons. Cell. ,vol. 115, pp. 135- 138 ,(2003) , 10.1016/S0092-8674(03)00760-8
Koenraad Devriendt, Annette S. Kim, Gert Mathijs, Suzanna G.M. Frints, Marianne Schwartz, Joost J. Van den Oord, Gregor E.G. Verhoef, Marc A. Boogaerts, Jean-Pierre Fryns, Daoqi You, Michael K. Rosen, Peter Vandenberghe, Constitutively activating mutation in WASP causes X-linked severe congenital neutropenia Nature Genetics. ,vol. 27, pp. 313- 317 ,(2001) , 10.1038/85886
RC Woodman, PE Newburger, P Anklesaria, RW Erickson, J Rae, MS Cohen, JT Curnutte, A new X-linked variant of chronic granulomatous disease characterized by the existence of a normal clone of respiratory burst-competent phagocytic cells. Blood. ,vol. 85, pp. 231- 241 ,(1995) , 10.1182/BLOOD.V85.1.231.BLOODJOURNAL851231
Xiaolin Wu, Yuan Li, Bruce Crise, Shawn M Burgess, Transcription start regions in the human genome are favored targets for MLV integration. Science. ,vol. 300, pp. 1749- 1751 ,(2003) , 10.1126/SCIENCE.1083413
U. P. Dave, Gene therapy insertional mutagenesis insights. Science. ,vol. 303, pp. 333- 333 ,(2004) , 10.1126/SCIENCE.1091667
P. Bousso, V. Wahn, I. Douagi, G. Horneff, C. Pannetier, F. Le Deist, F. Zepp, T. Niehues, P. Kourilsky, A. Fischer, G. de Saint Basile, Diversity, functionality, and stability of the T cell repertoire derived in vivo from a single human T cell precursor. Proceedings of the National Academy of Sciences of the United States of America. ,vol. 97, pp. 274- 278 ,(2000) , 10.1073/PNAS.97.1.274
K Pike-Overzet, D de Ridder, F Weerkamp, M R M Baert, M M A Verstegen, M H Brugman, S J Howe, M J T Reinders, A J Thrasher, G Wagemaker, J J M van Dongen, F J T Staal, Ectopic retroviral expression of LMO2, but not IL2Rγ, blocks human T-cell development from CD34+ cells: Implications for leukemogenesis in gene therapy Leukemia. ,vol. 21, pp. 754- 763 ,(2007) , 10.1038/SJ.LEU.2404563