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 ...
Jun 25, 2012
0
0
Investigators at Nationwide Children's have received a grant from the National Institutes of Health (NIH) to help move a therapy for MPS IIIB that has been shown effective in mice toward clinical trials in humans.
May 25, 2011
0
0
Many gene therapy strategies designed to deliver a normal copy of a gene to cells carrying a disease-causing genetic mutation rely on a modified virus to transfer the gene product into affected tissues. One technology platform ...
May 18, 2011
0
0
