Impact of Humoral Immune Response on Distribution and Efficacy of Recombinant Adeno-Associated Virus-Derived Acid -Glucosidase in a Model of Glycogen Storage Disease Type II
Cresawn K, Fraites T, Wasserfall C, Atkinson M, Lewis M, Porvasnik S, Liu C, Mah C, Byrne B. Impact of Humoral Immune Response on Distribution and Efficacy of Recombinant Adeno-Associated Virus-Derived Acid -Glucosidase in a Model of Glycogen Storage Disease Type II. Human Gene Therapy 2005, 16: 68-80. PMID: 15703490, DOI: 10.1089/hum.2005.16.68.Peer-Reviewed Original ResearchConceptsGlycogen storage disease type IIImmune-tolerant miceStorage disease type IIAntibody responseContractile functionImmune responseHumoral immune responseHind limb musclesAcid alpha-glucosidaseCardiorespiratory failureType IIAntibody formationReduced glycogenGlycogen reductionLysosomal storage diseaseSoleus muscleGAA levelsMiceEnzyme levelsSkeletal muscleStorage diseaseLevel of activityMuscleRecombinant adenoAlpha-glucosidase