Study reveals promising results for treating methylmalonic acidemia

A new study of methylmalonic acidemia (MMA) from NHGRI and University of Pennsylvania researchers found that most patients had a lower than expected immune reaction (indicated by the lack of antibodies) to a virus commonly used to deliver therapeutic genes. Gene therapy with the same virus was used to successfully treat mice with MMA. This means that most patients with MMA could be candidates for gene therapy. MMA is a rare and potentially life-threatening genetic disorder in which the body cannot properly turn food into energy.
Source: NHGRI Homepage Highlights - Category: Genetics & Stem Cells Source Type: news