Targeted delivery of therapeutic gene can effectively treat Pompe disease

Published on June 26, 2012 at 3:04 AM 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.(http://www.liebertpub.com) Continue reading