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Title of Journal: Biotechnol Lett

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Abbravation: Biotechnology Letters

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Springer Netherlands

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1573-6776

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Towards βglobin genetargeting with integrasedef

Authors: Davoud Nouri Inanlou Bagher Yakhchali Hossein Khanahmad Mossa Gardaneh Hesam Movassagh Reza Ahangari Cohan Mehdi Shafiee Ardestani Reza Mahdian Sirous Zeinali
Publish Date: 2010/07/17
Volume: 32, Issue: 11, Pages: 1615-1621
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Abstract

We have developed an integrasedefective lentiviral LV vector in combination with a genetargeting approach for gene therapy of βthalassemia The βglobin genetargeting construct has two homologous stems including sequence upstream and downstream of the βglobin gene a βglobin gene positioned between hygromycin and neomycin resistant genes and a herpes simplex virus type 1 thymidine kinase HSVtk suicide gene Utilization of integrasedefective LV as a vector for the βglobin gene increased the number of selected clones relative to nonviral methods This method represents an important step toward the ultimate goal of a clinical gene therapy for βthalassemiaAs gene therapy for βthalassemia using lentiviral vectors may not be acceptable because of unpredictable side effects identifying a solution that combines an efficient delivery of desired nucleic acids sequences with a homologous recombination mechanism has been pursued

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