A pilot study on using rapamycin-carrying synthetic vaccine particles (SVP) in conjunction with enzyme replacement therapy to induce immune tolerance in Pompe disease

Molecular Genetics and Metabolism Reports

Volumn 13, Issue , 2017, Pages 18-22

  Lim, Han Hyuk ^a   Yi, Haiqing ^a   Kishimoto, Takashi K ^b   Gao, Fengqin ^a   Sun, Baodong ^a   Kishnani, Priya S ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

^b Selecta Biosciences   (United States)

Author keywords

Acid alpha glucosidase; Enzyme replacement therapy; Pompe disease; Rapamycin; Tolerogenic nanoparticles

Indexed keywords

DRUG ANTIBODY; DRUG CARRIER; GLYCOGEN; METHOTREXATE; NANOPARTICLE; RAPAMYCIN; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; SYNTHETIC VACCINE PARTICLE; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DRUG UPTAKE; ENZYME REPLACEMENT; GLYCOGEN STORAGE DISEASE TYPE 2; IMMUNOLOGICAL TOLERANCE; MALE; MOTOR PERFORMANCE; MOUSE; NONHUMAN; PILOT STUDY; SKELETAL MUSCLE; WEIGHT REDUCTION;

EID: 85025168626     PISSN: None     EISSN: 22144269     Source Type: Journal    
DOI: 10.1016/j.ymgmr.2017.03.005     Document Type: Article

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