We previously determined the effective dose of rAAV9-dys3978 (PF-06939926), a recombinant AAV9 vector expressing a human mini-dystrophin gene under the control of a muscle-specific promoter, administered intravenously to 2 months old DMDmdx rats. An extensive natural history assessment of the DMDmdx rat model also reported a more aggressive phenotype than the mdx mouse model in both skeletal and cardiac muscle tissues, which progressed unfavourably with age. To understand the range of disease severity over which PF-06939926 has the potential to impact disease, we administered this gene therapy candidate to DMDmdx rats at 4 and 6 months of age.

Read More...

Leave a comment.

Your email address will not be published. Required fields are marked*

Andoird App
Loading...