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Treatment For Pompe Disease Enhanced By Targeted Gene Therapy

Gene therapy to replace the protein missing in Pompe disease can be effective if the patient’s immune system does not react against the therapy. Targeted delivery of the gene to the liver, instead of throughout the body, suppresses the immune response, improving the , according to an article published in , a peer-reviewed journal from Mary Ann Liebert, The article is available free online at the Human Gene Therapy website*.

“The current unmet medical need in Pompe disease is for prevention of immune responses against standard-of-care enzyme replacement therapy,” says coauthor , MD, PhD. “However, we foresee a future application of the dual strategy described in this paper, including a liver-expressing along with a ubiquitously expressing , which might achieve much higher efficacy than either alone.”

In the article “Immunodominant Liver-Specific Expression Suppresses Transgene-Directed Immune Responses in Murine Pompe Disease,” Ping Zhang and coauthors from Duke University Medical Center (Durham, NC), targeted a carrying the therapeutic gene to the livers of mice with Pompe disease. Not only did the liver-specific expression of the protein induce immune tolerance, but when combined with non-targeted delivery of the therapeutic gene it also boosted the overall effectiveness of the treatment.


Mary Ann Liebert, Inc./Genetic Engineering News