Novak Laboratory

About the Lab

Muscular dystrophies are genetic disorders characterized by muscle regenerative deficits, extensive muscle loss and fibrosis. One such disease, Duchenne muscular dystrophy (DMD), is a severe and relentlessly progressive myopathy that results from mutations in the X-linked DMD gene that disrupt the mRNA reading frame and prevent translation of the muscle structural protein, dystrophin. In DMD, the lack of dystrophin leads to chronic muscle degeneration, inflammation and fibrosis, resulting in a loss of muscle structural integrity and myofiber death. A promising genetic therapeutic strategy for DMD and other neuromuscular diseases is 'exon skipping' through the use of antisense oligonucleotides (AOs). This class of drug allows for ‘exon skipping’ of the mutated exons in the patient’s dystrophin gene to restore a functional, truncated dystrophin protein, and is currently the only approved genetic therapy for the treatment of DMD. A major focus of our research investigates how the complex pathological processes in DMD and muscular dystrophies modulate the delivery and therapeutic efficacy of antisense chemistries and gene therapies to develop novel delivery strategies to improve the pharmacokinetics and efficacy of these therapeutic agents for muscle diseases.

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  • Lab Focus Areas

    Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), Facioscapulohumeral muscular dystrophy (FSHD)
    Skeletal and cardiac muscle disease pathogenesis Therapeutic targets for muscular dystrophies
    Drug delivery strategies for muscle disease
    Muscle satellite cell dynamics during growth, aging and disease

  • Partnership

    George Washington University School of Medicine and Health Sciences

  • Contact

    James Novak, Ph.D. Principal Investigator 202-476-6135

Highlights from the Lab

  • Internship Opportunities

    Our laboratory is committed to training and mentoring the next generation of young scientists.

  • News

    Experimental model mimics early-stage myogenic deficit in boys with DMD.