Why the study?
Does retargeting dual hcAd/AAV hybrid vectors with Ad50 fiber domains improve dystrophin synthesis in DMD muscle cells compared to conventional Ad5 fibers?
Population
Muscle cells (myoblasts and myotubes) from Duchenne muscular dystrophy (DMD) patients
Comparison
Dual high-capacity adenovirus-adeno-associated… vs Isogenic dual hcAd/AAV hybrid vector particles…
Design
Preclinical
Authors
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Ad50-retargeted vectors may enhance ex vivo DMD gene therapy; leaves open in vivo efficacy and clinical translation.
Does retargeting dual hcAd/AAV hybrid vectors with Ad50 fiber domains improve dystrophin synthesis in DMD muscle cells compared to conventional Ad5 fibers?
Retargeting dual hcAd/AAV hybrid vectors with Ad50 fiber domains enables CAR-independent uptake and robust dystrophin synthesis in DMD muscle cells, offering a potential improvement for ex vivo gene therapy.
Gonçalves et al. (2006) studied this question.
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