Update on clinical gene therapy in childhood

Qasim, Waseem; Gaspar, H. Bobby; Thrasher, Adrian J.
November 2007
Archives of Disease in Childhood;Nov2007, Vol. 92 Issue 11, p1028
Academic Journal
The successful use of gene therapy to correct rare immune system disorders has highlighted the enormous potential of such therapies. We review the current state of gene therapy for childhood immune system disorders, and consider why these conditions have been particularly amenable to genetic correction. As with all emerging therapies, there have been unexpected side effects and their underlying mechanisms are the subject of intense research. Minimising such risks through improved vector design will play an important role in developing the next generation of gene based therapies and extending their applicability.


Related Articles

  • Gene Therapy Cures Form of "Bubble Boy Disease".  // Cryonics;1st Quarter 2009, Vol. 30 Issue 1, p21 

    The article reports on the research on the gene therapy for the treatment of bubbly boy disease that was published in the January 29, 2009 issue of the journal 'New England Journal of medicine." The study followed the progress of the patients involved in it for about four years after treatment....

  • ZAP-70 Restoration in Mice by In Vivo Thymic Electroporation. Irla, Magali; Saade, Murielle; Kissenpfennig, Adrien; Poulin, Lionel Franz; Leserman, Lee; Marche, Patrice N.; Jouvin-Marche, Evelyne; François Berger; Nguyen, Catherine // PLoS ONE;2008, Vol. 3 Issue 4, p1 

    Viral and non-viral vectors have been developed for gene therapy, but their use is associated with unresolved problems of efficacy and safety. Efficient and safe methods of DNA delivery need to be found for medical application. Here we report a new monopolar system of non-viral electro-gene...

  • severe combined immunodeficiency (SCID):. Huber, Jeffrey T.; Gillaspy, Mary L. // Encyclopedic Dictionary of AIDS-Related Terminology;2000, p198 

    A definition of the term "severe combined immunodeficiency" is presented. It refers to a group of rare congenital disorders characterized by impairment of both humoral and cell-mediated immunity manifested by lack of antibody formation in response to the presence of antigens, lack of delayed...

  • SCID. Peters, Michael // BMA A-Z Family Medical Encyclopedia;2004, p676 

    An encyclopedia entry for the abbreviation "SCID," which refers to severe combined immunodeficiency, is presented.

  • A Case of IL-7R Deficiency Caused by a Novel synonymous Mutation and Implications for Mutation screening in sCID Diagnosis. Gallego-Bustos, Fernando; Gotea, Valer; Ramos-Amador, José T.; Rodríguez-Pena, Rebeca; Gil-Herrera, Juana; Sastre, Ana; Delmiro, Aitor; Rai, Ghadi; Elnitski, Laura; Gonzalez-Granado, Luis I.; Allende, Luis M. // Frontiers in Immunology;10/27/2016, Vol. 7, p1 

    Reported synonymous substitutions are generally non-pathogenic, and rare pathogenic synonymous variants may be disregarded unless there is a high index of suspicion. In a case of IL7 receptor deficiency severe combined immunodeficiency (SCID), the relevance of a non-reported synonymous variant...

  • 20 years of gene therapy for SCID. Fischer, Alain; Hacein-Bey-Abina, Salima; Cavazzana-Calvo, Marina // Nature Immunology;Jun2010, Vol. 11 Issue 6, p457 

    In this article, the author discusses the effectiveness of gene therapy for the treatment of severe combined immunodeficiency (SCID). He mentions that the absence of the transplantation of allogeneic hematopoietic stem cells (HSCs) has driven the emergence of the idea of gene therapy in which...

  • THE DNA CURE. Neimark, Jill // Discover;Sep2009, Vol. 30 Issue 8, p36 

    The article discusses the reemergence of gene therapy as a result of its success in the treatments for blindness, cancer, and other immune diseases. The focus of the article is a young girl named Katlyn Demerchant who was diagnosed with severe combined immunodeficiency (SCID), whose treatment...

  • Maternal effects of the scid mutation on radiation-induced transgenerational instability in mice. Hatch, T.; Derijck, A. A. .H .A.; Black, P. D.; van der Heijden, G. W.; de Boer, P.; Dubrova, Y. E. // Oncogene;7/12/2007, Vol. 26 Issue 32, p4720 

    The results of a number of recent studies show that mutation rates in the offspring of irradiated parents are substantially elevated, however, the effect of parental genotype on transgenerational instability remains poorly understood. Here, we have analysed the mutation frequency at an expanded...

  • Animal Ca2+ release-activated Ca2+ (CRAC) channels appear to be homologous to and derived from the ubiquitous cation diffusion facilitators. Matias, Madeleine G.; Gomolplitinant, Kenny M.; Tamang, Dorjee G.; Saier, Jr, Milton H. // BMC Research Notes;2010, Vol. 3, p158 

    Background: Antigen stimulation of immune cells triggers Ca2+ entry through Ca2+ release-activated Ca2+ (CRAC) channels, promoting an immune response to pathogens. Defects in a CRAC (Orai) channel in humans gives rise to the hereditary Severe Combined Immune Deficiency (SCID) syndrome. We here...


Read the Article


Sorry, but this item is not currently available from your library.

Try another library?
Sign out of this library

Other Topics