
AAV2/8 mediates sustained transgene expression in the mouse abdomen and thorax.
Researchers have developed a new strategy using lung-targeted gene therapy that may lead to improved treatments for inherited diseases including emphysema. Alpha-1-antitrypsin deficiency (AATD) is the most common genetic cause of emphysema, a chronic lung disease that leads to significant morbidity and mortality. AATD, which affects up to 100,000 Americans, is caused by inheritance of a single gene mutation.
The current treatment for patients affected by AATD involves weekly intravenous infusion of the normal AAT protein – an inconvenient, invasive and expensive option. Delivery of a normal copy of the gene, known as gene therapy, is an experimental approach to treating some genetic conditions including AATD...
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