Long-Term Functional Correction of Pompe Disease and Increased α-Glucosidase Expression after Gene Therapy with Novel Combinations of Muscle-Targeted Transcriptional Cis -Regulatory Elements

Abstract excerpt

The development of efficient and safe muscle-directed gene therapy is an unmet medical need. One of the bottlenecks in muscle-directed gene therapy is the high levels of muscle-targeted transcription required in these afflicted target tissues. To circumvent this problem, novel transcriptional cis-regulatory elements (CREs) were identified by transcriptome-wide data-mining that led to a significant increase of...

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