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Gene Therapy Effective For Pompe Disease In Mice Prepared For Human Trials: Study

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Duke Health researchers have developed a gene therapy that could enhance or even replace the only FDA-approved treatment currently available to patients with a rare, life-threatening condition that cripples the muscles. The condition known as Pompe disease is inheritable and affects around 1 in every 20,000 babies and can also affect adults.

The gene therapy used in mice utilizes a modified virus to send a gene, which produces GAA to the liver – an enzyme people with Pompe disease do not have. The study authors have received approval from the FDA to conduct a Phase 1 clinical trial in humans and are now working to secure funding for the research.

People with the condition do not have the GAA enzyme, which invariably means their bodies will not be able to breakdown sugar, resulting in the buildup of glycogen in the muscles. The abnormal buildup causes improper muscle development in babies and can lead also to respiratory problems, heart failure and even death, if left undiagnosed and untreated.

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