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10.3233/TRD-160007

http://scihub22266oqcxt.onion/10.3233/TRD-160007
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C5108627!5108627 !27853673
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suck abstract from ncbi


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pmid27853673
      Transl+Sci+Rare+Dis 2016 ; 1 (1 ): 73-89
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  • Gene Therapy for Metabolic Diseases #MMPMID27853673
  • Chandler RJ ; Venditti CP
  • Transl Sci Rare Dis 2016[]; 1 (1 ): 73-89 PMID27853673 show ga
  • Gene therapy has recently shown great promise as an effective treatment for a number of metabolic diseases caused by genetic defects in both animal models and human clinical trials. Most of the current success has been achieved using a viral mediated gene addition approach, but gene-editing technology has progressed rapidly and gene modification is being actively pursued in clinical trials. This review focuses on viral mediated gene addition approaches, because most of the current clinical trials utilize this approach to treat metabolic diseases.
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