Gene Therapy for Erythroid Metabolic Inherited Diseases
نویسندگان
چکیده
Gene therapy is becoming a powerful tool to treat genetic diseases. Clinical trials performed during last two decades have demonstrated its usefulness in the treatment of several genetic diseases [1] but also the need to improve vector delivery, expression and safety [2]. New vectors should reduce genotoxicity (genomic alteration due to vector integration), immuno‐ genicity (immune response to gene delivery vectors and/or trangenes) and cytotoxicity (in‐ duced by ectopic expression and/or overexpression of the transgene).
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تاریخ انتشار 2013