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10.1089/hum.2014.102

http://scihub22266oqcxt.onion/10.1089/hum.2014.102
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C4179920!4179920!25184354
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suck abstract from ncbi


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pmid25184354      Hum+Gene+Ther 2014 ; 25 (10): 862-5
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  • Lentiviral Hematopoietic Stem Cell Gene Therapy in Inherited Metabolic Disorders #MMPMID25184354
  • Wagemaker G
  • Hum Gene Ther 2014[Oct]; 25 (10): 862-5 PMID25184354show ga
  • After more than 20 years of development, lentiviral hematopoietic stem cell gene therapy has entered the stage of initial clinical implementation for immune deficiencies and storage disorders. This brief review summarizes the development and applications, focusing on the lysosomal enzyme deficiencies, especially Pompe disease.
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