Efficacy of gene therapy for SCID is being confirmed

Département de Biothérapie, Hôpital Necker-Enfants Malades, 75015 Paris, France.
The Lancet (Impact Factor: 45.22). 12/2004; 364(9452):2155-6. DOI: 10.1016/S0140-6736(04)17603-4
Source: PubMed
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    • "Because the common γ-chain is shared by many cytokine receptors (receptors for IL-2, IL-4, IL-7, IL-9, IL-15, and IL-21), mutations that result in a non-functional common γ-chain cause widespread defects in IL signaling and failure of B and T cells to develop and mature. This is a rather profound example, and knowledge of the responsible gene has opened avenues for therapeutic intervention in cases of X-linked SCID, where gene transfer of the normal CD132 appears to be a promising, even curative, treatment modality [4,5]. Similar observations and clinical conclusions have been made with another form of SCID caused by defects in the variable, diversity, joining recombination process [6]. "
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    ABSTRACT: The recent advent of genomic approaches for association testing is starting to enable a more comprehensive understanding of the role of human immune response in determining infectious disease outcomes. Progressing from traditional linkage approaches using microsatellite markers to high-resolution genome-wide association scans, these new approaches are leading to the robust discovery of a large number of disease susceptibility genes and the beginnings of an appreciation of their connections. In this commentary, we discuss how this technology development has led to increasingly complex and common infectious diseases being unraveled, and how this is starting to dissect pathogen-specific human responses. Intriguingly, these still preliminary findings suggest that pathogen innate detection mechanisms may not be as shared among diseases as immune response mechanisms.
    Genome Medicine 08/2010; 2(8):52. DOI:10.1186/gm173 · 5.34 Impact Factor
    • "Indeed, gene therapy has proven over the past years to be a solution for several inherited diseases such as severe combined immunodeficiency (SCID) [1] [2], adenosine deaminase deficiency (ADA) [3] and hemophilia [4]. Very recently, a gene therapy trial of chronic granulomatous disease (CGD) resulted in successful treatment of the myeloid compartment [5] [6]. "
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    ABSTRACT: Vectors derived from retroviruses such as lentiviruses and onco-retroviruses are probably among the most suitable tools to achieve a long-term gene transfer since they allow stable integration of a transgene and its propagation in daughter cells. Lentiviral vectors should be preferred gene delivery vehicles over vectors derived from onco-retroviruses (MLV) since in contrast to the latter they can transduce non-proliferating target cells. Moreover, lentiviral vectors that have the capacity to deliver transgenes into specific tissues are expected to be of great value for various gene transfer approaches in vivo. Here we provide an overview of innovative approaches to upgrade lentiviral vectors for tissue or cell targeting and which have potential for in vivo gene delivery. In this overview we distinguish between three types of lentiviral vector targeting strategies (Fig 1): 1) targeting of vectors at the level of vector-cell entry through lentiviral vector surface modifications; 2) targeting at the level of transgene transcription by insertion of tissue specific promoters into lentiviral vectors; 3) a novel microRNA technology that rather than targeting the 'right' cells will 'detarget' transgene expression from non-target cells while achieving high expression in the target-cell. It is clear that each strategy is of enormous value for several gene therapy approaches but combining these three layers of transgene expression control will offer tools to really overcome several drawbacks in the field such as side-effect of off-target expression, clearance of transgene modified cells by immune response to the transgene and lack of biosecurity and efficiency in in vivo approaches.
    Current Gene Therapy 01/2009; 8(6):449-60. DOI:10.2174/156652308786848003 · 2.54 Impact Factor
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    ABSTRACT: Incluye resumen y conclusiones en inglés Tesis Univ. Granada. Departamento de Bioquímica y Biología Molecular. Leída el 26 de junio de 2007
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