A new study in patients with the inherited metabolic disorder methylmalonic academia (MMA) found lower than expected levels of antibodies against the adeno-associated viral (AAV) vectors being developed for gene therapy to replace the enzyme (MUT) that is deficient in MMA. These results, and especially the absence of AAV antibodies in most patients who completely lack the MUT enzyme and have not had a liver transplant—those in greatest need of improved therapies—has encouraging implications for gene delivery as a treatment for MMA, as described in an article in Human Gene Therapy.
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