January 2, 2017Human Gene TherapyOpen Access

Comparison of Serum rAAV Serotype-Specific Antibodies in Patients with Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Inclusion Body Myositis, or GNE Myopathy

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Structured PICO

Population

100 human subjects including patients with Duchenne muscular dystrophy (n=22), Becker muscular dystrophy (n=16), inclusion body myositis (n=18), GNE myopathy (n=4), young normal subjects <18 years (n=19), and adult normal subjects >18 years (n=21). Also includes 9 serum-naïve macaques.

Comparator

Normal human subjects (young and adult)

Outcome

Pre-existing serum antibody titers to rAAVrh74, rAAV1, rAAV2, rAAV6, rAAV8, and rAAV9surrogate

Most DMD and BMD patients have low or no pre-existing serum antibodies to rAAVrh74, suggesting they are amenable to vascular rAAV-mediated gene therapy without treatment blockage.

Discussion

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Implication

Lower seroprevalence in DMD/BMD may support rAAV gene therapy eligibility; leaves open optimal serotype screening in trials.

Cite This Study

Zygmunt et al. (2017) studied this question.

synapsesocial.com/papers/6a835fa5be60e97fec18fdcd https://doi.org/10.1089/hum.2016.141

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Comparison of Serum rAAV Serotype-Specific Antibodies in Patients with Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Inclusion Body Myositis, or GNE Myopathy

Structured PICO

Discussion

Implication

Cite This Study

Comparison of Serum rAAV Serotype-Specific Antibodies in Patients with Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Inclusion Body Myositis, or GNE Myopathy

Structured PICO

Discussion

Implication

Cite This Study

Also Consider

Also Consider