Gene Therapy For Alpha-1 Antitrypsin Deficiency

Filed in archive Diagnostics, Methodologies and Instrumentation , Drugs, Vaccines and Therapeutics , Genomics, Proteomics and Bioinformatics on November 22, 2006

A clinical trial evaluating the safety and efficacy of using gene therapy to treat alpha-1 antitrypsin deficiency, a hereditary lung and liver disease, showed promising results. The researchers used an adeno-associated virus to deliver a gene coding for alpha-1 antitrypsin, an anti-inflammatory protein.

"We found that we can use this agent safely and we also saw evidence in the patients' blood that the higher doses successfully introduced the vector DNA. In one patient we saw evidence for a very brief period that some of the alpha-1 protein was being produced, but not at a high enough level to be beneficial."

Succeeding trials aim to test the therapy with a different version of the adeno-associated virus which showed more potency in animal trials. The results of the abovementioned study were published online in Human Gene Therapy.

[Image: ADUK]