Blood Advances · 2018 · 22 citations · 25 references
Life SciencesHematologyImmunologyGene TherapyImmunogeneticsImmunoregulationImmune RegulationImmunogenicity Of Gene TherapiesImmunological Disease ModelsEditing For Inherited Blood DisordersGene CorrectionImmune Tolerance AssessmentImmune Tolerance FailureImmune Tolerance RestorationMuscle Gene TherapyMuscle Regeneration Gene Therapy
Key Points Skeletal muscle–directed expression of FIX-Padua resulted in complete correction of HB phenotype in an inhibitor-prone dog model. Long-term immune tolerance to FIX is sustained over years upon multiple challenges with recombinant FIX protein in 2 HB models.
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Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
Amit Nathwani, Ulreke M. Reiss, Edward G. D. Tuddenham et al. · New England Journal of Medicine · 2014 · 1.3K citations · Full text
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
Catherine S. Manno, Ah Keng Chew, S Hutchison et al. · Blood · 2003 · 747 citations · Full text
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Dystrophin Immunity in Duchenne's Muscular Dystrophy
Jerry R. Mendell, Katherine Campbell, Louise R. Rodino‐Klapac et al. · New England Journal of Medicine · 2010 · 655 citations · Full text