458. Development of Non-Integrating and Site- Specifically Integrating Lentiviral Vectors
نویسندگان
چکیده
منابع مشابه
Design and Potential of Non-Integrating Lentiviral Vectors
Lentiviral vectors have demonstrated promising results in clinical trials that target cells of the hematopoietic system. For these applications, they are the vectors of choice since they provide stable integration into cells that will undergo extensive expansion in vivo. Unfortunately, integration can have unintended consequences including dysregulated cell growth. Therefore, lentiviral vectors...
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BACKGROUND The efficacy and biosafety of lentiviral gene transfer is influenced by the design of the vector. To this end, properties of lentiviral vectors can be modified by using cis-acting elements such as the modification of the U3 region of the LTR, the incorporation of the central flap (cPPT-CTS) element, or post-transcriptional regulatory elements such as the woodchuck post-transcriptiona...
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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 ...
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The main objective of an ecosystem sustainable management is to preserve itscapacity to respond and adapt to current disturbances and/or future changes, and maintain theprovision of environmental goods and services. Two very important properties linked to thisobjective are the ecosystem resilience and resistance to disturbance factors. The objective ofthis paper is to recommend conceptual modif...
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Currently the best algorithms for transcription factor binding site prediction are severely limited in accuracy. There is good reason to believe that predictions from these different classes of algorithms could be used in conjunction to improve the quality of predictions. In this paper, we apply single layer networks, rules sets and support vector machines on predictions from 12 key algorithms....
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ژورنال
عنوان ژورنال: Molecular Therapy
سال: 2006
ISSN: 1525-0016
DOI: 10.1016/j.ymthe.2006.08.527