Open AccessAssessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease
Author(s) -
Gensheng Wang,
Sarah P. Young,
Deeksha Bali,
Julie A. Hutt,
Songtao Li,
Janet M. Benson,
Dwight D. Koeberl
Publication year - 2019
Publication title -
molecular therapy — methods and clinical development
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.285
H-Index - 32
ISSN - 2329-0501
DOI - 10.1016/j.omtm.2019.05.001
Subject(s) - genetic enhancement , recombinant dna , vector (molecular biology) , toxicity , biodistribution , enzyme replacement therapy , viral vector , medicine , pharmacology , virology , virus , disease , immunology , biology , gene , in vivo , biochemistry , microbiology and biotechnology
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