Understanding obstacles to retroviral mediated gene therapy of globin disorders
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Understanding obstacles to retroviral mediated gene therapy of globin disorders implications for design of therapeutic expresssion[sic] cassettes and retrovirus vectors. by Dylan Geoffrey Pannell

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Published .
Written in English


Book details:

The Physical Object
Pagination248 leaves.
Number of Pages248
ID Numbers
Open LibraryOL19346864M
ISBN 100612691926

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  Globin Gene Therapy Vectors: – In the mid s, the locus control region (LCR) for the β-globin gene cluster was defined as a series of nuclease hypersensitivity (HS) sites in the 21 kb of DNA upstream from the β-globin locus (10, 11).Functional studies in transgenic mice demonstrated that the LCR possessed powerful erythroid-specific enhancer activity ().Cited by: Regulated expression of the human beta-globin gene has been demonstrated in cultured murine erythroleukemia cells and in mice after retrovirus-mediated gene transfer. Indeed, this review was prompted by our observation of a leukemia in a mouse study with prolonged follow-up after retroviral gene transfer into hematopoietic cells Unfortunately, the first case of a malignant disorder following clinical retroviral vector-mediated gene transfer into human hematopoietic cells was observed shortly thereafter Cited by: In book: A Guide to Human Gene Therapy, pp Retroviral-mediated gene transfer of human beta-globin provides a model system for the development of somatic gene therapy for.

These phenomena were observed to various degrees in mouse transplant experiments using retroviral vectors containing a human β-globin gene, even when cis-linked to locus control region derivatives.   Several decades ago, the first retroviral vectors were constructed. They have been proved as delivery vehicles in basic and translational research; many of them were used in clinical trials in the treatment of genetic and immunologic disorders or malignancies to deliver therapeutic genes into target tissue. Gammaretroviral and lentiviral vectors are popular viral delivery vehicles; their.   Gene therapy is a novel promising approach for treating a spectrum of inherited and non-inherited disorders by delivering therapeutic genes to specific organs or tissues. Of the viral vectors that have been used to date to deliver the genes of interest, the adeno-associated viral (AAV) vector appears to be the most safe and effective vehicle and has the ability to maintain long-term gene and. Human Gene Therapy for Retinal Disorders. A thorough understanding of the natural history of a disease is an important element in all clinical development programs. Many degenerative retinal.

  This is the main advantage of RNAi-mediated gene therapy over drug therapy. Improved understanding of molecular interactions in fetal-to-adult hemoglobin switching mechanism holds the key to the identification of novel therapeutic targets not only for SCD and β-thalassemia but also for other β-globin disorders.   Toward gene therapy for disorders of globin synthesis but this approach has been impeded by formidable obstacles over the last decade. (suppl 1, abstr) Karlsson S, Bodine DM, Perry L, et al: Expression of the human beta-globin gene following retroviral-mediated transfer into multipotential hematopoietic progenitors of mice. Proc.   Introduction. Hemoglobin (Hb), the oxygen-carrying molecule, contains two identical α- and β-globin chains linked to a heme group ().ß-globinopathies are inherited disorders characterized by mutations that decrease the synthesis of the ß-globin chain, as in ß-thalassemia, or lead to a mutated ß-globin variant, as in sickle cell disease (SCD) (β S), which causes hemoglobin polymerization. Therapy for Globin-Chain Disorders To the Editor: The clinical evaluation of treatments for disorders of globin-chain synthesis has entered an exciting new era.