Targeted gene therapy enhances treatment for Pompe disease

June 25, 2012

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 therapeutic effect, according to an article published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc. 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 ," says coauthor Dwight Koeberl, MD, PhD. "However, we foresee a future application of the dual vector strategy described in this paper, including a liver-expressing vector along with a ubiquitously expressing vector, which might achieve much higher efficacy than either vector 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 gene delivery vector carrying the therapeutic gene to the livers of mice with Pompe disease. Not only did the liver-specific expression of the protein induce , but when combined with non-targeted delivery of the therapeutic gene it also boosted the overall effectiveness of the treatment.

Explore further: New study shows that therapeutic gene expression can be sustainable for 1 year

More information: DOI: 10.1089/hum.2011.063

Related Stories

Cancer drugs help the hardest cases of Pompe disease

January 5, 2012

Kids with Pompe disease fail because of a missing enzyme, GAA, that leads to dangerous sugar build-up, which affects muscles and movement. An enzyme replacement treatment pioneered at Duke University has saved many lives, ...

Recommended for you

Zika virus may persist in the vagina days after infection

August 25, 2016

The Zika virus reproduces in the vaginal tissue of pregnant mice several days after infection, according to a study by Yale researchers. From the genitals, the virus spreads and infects the fetal brain, impairing fetal development. ...

In sub-Saharan Africa, cancer can be an infectious disease

August 26, 2016

In 1963, Irish surgeon Denis Parson Burkitt airmailed samples of an unusual jaw tumor found in Ugandan children to his colleague, Anthony Epstein, at Middlesex Hospital in London. Epstein, an expert in chicken viruses and ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.