A team of researchers at the University of Texas Southwestern Medical Center has developed an innovative gene-editing delivery system that can target multiple organs at the same time. This breakthrough could transform the treatment of various genetic diseases, including a rare condition known as alpha-1 antitrypsin deficiency (AATD).
The new system uses specially designed lipid nanoparticles to deliver gene-editing tools directly to the liver and lungs, addressing both areas affected by AATD. This genetic disorder occurs due to a single mutation in the SERPINA1 gene, which leads to the accumulation of a toxic protein in the liver and can cause damage to the lungs, leading to conditions like emphysema.
Dr. Daniel Siegwart, a professor at the medical center and the leader of this research, noted, “Many diseases affect more than one organ. Developing therapies that can target multiple organs opens new doors for treatment.”
Previously, the research team developed a method called Selective Organ Targeting (SORT), which allowed them to direct gene-editing molecules to specific organs. However, this recent advancement is significant because it demonstrates the ability to target both the liver and lungs simultaneously.
In the study published in the journal Nature Biotechnology, the researchers conducted tests on mice genetically modified to carry the human gene mutation associated with AATD. After a single dose of their new delivery system, they achieved gene editing in about 40% of liver cells and 10% of lung cells, directly reducing the levels of the harmful protein in both organs.
The results were promising. Levels of the mutated protein in the liver decreased by 80%, and signs of lung enzyme damage were reduced by 89% within weeks of treatment. This illustrates not only the effectiveness of the gene-editing process but also the potential for long-lasting improvements in the health of patients with similar genetic disorders.
The implications of this research extend beyond AATD. The ability to create therapies that reach multiple organs means that there is hope for more comprehensive treatments for other multi-organ diseases as well.
Dr. Siegwart and his team are working toward turning their research findings into clinical therapies through a company they co-founded called ReCode Therapeutics, which is focused on further developing these innovative gene-editing applications.
-Rashmi kumari
