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Clinical Trials


Duchenne Muscular Dystrophy Clinical Trial

The first U.S. gene therapy clinical trial for Duchenne Muscular Dystrophy launched in March 2006. Dr. Samulski collaborated with colleagues at Ohio State University and the University of Pittsburgh to develop and produce an AAV vector containing a functional miniaturized dystrophin gene.


Stilwell J and Samulski RJ. 2004. Role of Viral Vectors and Virion Shells on Cellular Gene Expression. Molecular Therapy, 9(3):337-346.

This study focuses on the role of the viral capsid on cellular gene expression, and indicates that its role must be considered independent of the vector genome. DNA microarray technology compared the effects of AAV and adenovirus. AAV had minimal impact on gene expression, while adenovirus exposure activated immune and stress responses. Therefore, AAV may be a safer, more effective method of transduction. These findings have great potential to improve gene therapy safety in the clinic. Drs. Samulski and Stilwell have received recognition from the scientific community and the press for their accomplishments.

  • News and Views commentary on this study - Weitzman, MD. 2004. Vector-omics. Molecular Therapy, 9(3):315-317.


Canavan Disease Clinical Trial

The fall 2003 edition of UNC's Endeavors magazine documents Dr. Samulski's UNC Gene Therapy Clinical Trialscollaboration with Paola Leone, PhD. Dr. Leone is an associate professor at the Robert Wood Johnson Medical School in Camden, NJ. Together, they are working on a gene therapy treatment for Canavan disease, a fatal neurodegenerative disorder that affects children. This disease is caused by a single defective gene, which results in abnormal brain development and function. There are no drugs or treatments for the disease, and children with the genetic defect die at an early age.

Canavan disease is a suitable target for gene therapy because the genetic abnormality is limited to a single gene. Our study attempts to replace the defective gene with a flawless copy, which becomes one with the child's DNA. Dr. Samulski's lab produces the gene based drug and colleagues at RWJMS-Camden administer this drug to subjects in a clinical trial. To date, more than seven children have received the treatment, and studies are in progress to determine the outcome of this experimental DNA drug. More information related to Canavan and gene therapy based treatment can be found in the below article.

Click here to download the Endeavors article (PDF)
This article, written by Anton Zuiker, originally appeared in the Fall 2003 edition of Endeavors Magazine



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