Literature DB >> 17631450

[Cell therapy for inherited diseases of the hematopoietic system].

Marina Cavazzana-Calvo1, Liliane Dal-Cortivo, Isabelle André-Schmutz, Salima Hacein-Bey Abina, Alain Fischer.   

Abstract

Cell therapy was born in 1968 with the first allogeneic transplantation of hematopoietic stem cells for two immune deficiency disorders: the Wiskott-Aldrich syndrome and the Severe Combined ImmunoDeficiency (SCID). From this pioneering experience, thousands of patients affected with inherited or acquired diseases of the hematopoietic system have benefited from this therapeutic approach. Unfortunately, immunologic obstacles, represented by the compatibility in the major histocompatibility HLA system, still dictate today important limitations for a larger therapeutic utilization of hematopoietic stem cells (HSC). In this review, we have summarized the difficulties and the scientific advances leading us to improve the clinical results; the therapeutic research's track for primary immunodeficiencies is also discussed.

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Year:  2007        PMID: 17631450     DOI: 10.1016/j.crvi.2007.04.003

Source DB:  PubMed          Journal:  C R Biol        ISSN: 1631-0691            Impact factor:   1.583


  1 in total

1.  Getting the Most: Enhancing Efficacy by Promoting Erythropoiesis and Thrombopoiesis after Gene Therapy in Mice with Hurler Syndrome.

Authors:  Jing-Fen Han; Salim S El-Amouri; Mei Dai; Phuong Cao; Dao Pan
Journal:  Mol Ther Methods Clin Dev       Date:  2018-10-10       Impact factor: 6.698

  1 in total

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