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Accession Number:
AD1197401
Title:
Novel Therapeutic Strategy to Achieve Upregulation of Dystrophin Isoforms
Report Date:
2022-10-01
Abstract:
The objective of this project is to achieve a single-vector CRISPR-dCas9 gene activation platform to upregulate near full-length muscle dystrophin isoform (Dp427m) as a therapeutic strategy to compensate for its reduction in candidate dystrophinopathy patients with in-frame deletions. Our lead construct design will be systemically delivered using AAV9 and tested for the optimal dose required to achieve therapeutic upregulation of the dystrophin gene from its endogenous locus in both skeletal and cardiac muscles. A transgenic humanized DMD/BMD mousemodel harboring a deletion of exon 45-49 of muscle dystrophin will be generated to enable functional assessment of our construct and the therapeutic potential of upregulating truncated dystrophin.
Document Type:
Conference:
Journal:
Pages:
13
File Size:
3.06MB
W81XWH-21-1-0956
(W81XWH2110956 );
Contracts:
Grants:
Distribution Statement:
Approved For Public Release
