منابع مشابه
Beta thalassemia gene therapy using lentiviral vectors
Recent years, allogeneic bone marrow transplantation (BMT) has proved to be the successful cure for patients with thalassemia major, however this is restricted due to limited matched-related donor. Its complications include chronic graft-versus-host disease in 5-8% of patients. So, a molecular approach, such as gene therapy for direct normal beta globin gene transmission, seems quite promising ...
متن کاملLentiviral vectors and gene therapy.
Gene therapy is a novel method under investigation for the treatment of genetic, metabolic and neurologic diseases, cancer and AIDS. The primary goal of gene therapy is to deliver a specific gene to a pre-determined target cell, and to direct expression of such a gene in a manner which will result in a therapeutic effect. Retroviral vectors have the ability to integrate in the host cell DNA irr...
متن کاملLentiviral Vectors and Cystic Fibrosis Gene Therapy
Cystic fibrosis (CF) is a chronic autosomic recessive syndrome, caused by mutations in the CF Transmembrane Conductance Regulator (CFTR) gene, a chloride channel expressed on the apical side of the airway epithelial cells. The lack of CFTR activity brings a dysregulated exchange of ions and water through the airway epithelium, one of the main aspects of CF lung disease pathophysiology. Lentivir...
متن کاملHIV-Derived Lentiviral Vectors: Current Progress toward Gene Therapy and DNA Vaccination
Lentiviral vectors are promising gene delivery tools capable of transducing a variety of dividing and non-dividing cells, including pluripotent stem cells which are refractory for transduction by murine retroviruses. Although there is a growing debate on the safety of lentiviral vectors for gene transfer, in particular for those derived from human immunodeficiency viruses, type one (HIV-1) and ...
متن کاملLentiviral Vectors: Not Just For Gene Therapy
Lentiviral vectors (LVs) are viral-based gene delivery systems that can stably deliver genes or RNAi into primary cells or cell lines with up to 100% efficiency. LVs bind to target cells using an envelope protein which allows for release of the LV RNA containing the gene or gene silencing sequence into the cell. The LV’s RNA is then converted into DNA using an enzyme called reverse transcriptas...
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ژورنال
عنوان ژورنال: Frontiers in Bioscience
سال: 1999
ISSN: 1093-9946,1093-4715
DOI: 10.2741/klimatcheva