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The Children's Research Institute

James Novak, Ph.D., Laboratory

Novak Lab

Antisense Oligonucleotide Delivery

Our current research focuses on defining the mechanisms of delivery for different antisense oligonucleotide chemistries.

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Duchenne muscular dystrophy (DMD) is a severe and relentlessly progressive myopathy which results from out-of-frame or nonsense mutations in the X-linked DMD gene that disrupt the mRNA open reading frame and prevent translation of dystrophin protein. In DMD, the lack of dystrophin leads to persistent muscle degeneration and regeneration, inflammation, and fibrosis, resulting in a loss of myofiber structural integrity and functional strength. A promising therapeutic strategy for DMD is 'exon skipping', employing antisense oligonucleotides to exclude disruptive exons from mRNA transcript, thus permitting translation of a truncated, partially functional dystrophin protein.

Our research directions focus on investigating how pathological features of DMD modulate the delivery and therapeutic efficacy of different antisense chemistries in the context of genetic background and disease pathology through of use of different mouse and cell culture-based systems. The long-term goals of our research are to optimize exon skipping treatment strategies for clinical application and identify novel therapeutic targets to improve this promising approach for DMD.

James Novak, Ph.D., Laboratory

James Novak

Duchenne muscular dystrophy (DMD) is a severe and relentlessly progressive myopathy which results from out-of-frame or nonsense mutations in the X-linked DMD gene that disrupt the mRNA open reading frame and prevent translation of dystrophin protein.