New gene-editing tech holds promise for treating complex genetic diseases

There are researchers that are currently working at the University of Texas and are trying to find more effective methods to treat genetic disorders. They are using bacterial elements called retrons, with a new system in place that can repair multiple mutations at once instead of the traditional one. This is incredibly helpful, especially for diseases such as cystic fibrosis which have many of the same mutations within one gene. The retron based method is known to work effectively, while adjusting cells by 30%. By adjusting the large defective sections of DNA with healthier ones, this could lead to more inclusive and cheaper gene therapies. A graduate student from UT said “My hope, and what drives me, is to develop a gene-editing technology that’s much more inclusive of people who might have more unique disease-causing mutations, and that using retrons will be able to expand that impact onto a much broader patient population” (New gene-editing tech holds promise for treating complex genetic diseases, 2025).

The new method is giving researchers hope since it can replace a large quantity of the defective DNA in a healthy sequence. This can also fix any type of combination of mutations in the DNA strand without having to “be specific to any one person’s genetics”. This discovery is a huge step towards better genetic treatment options. This offers help for individuals with really rare mutations who are usually left with little to no effective options. 

 

Gene therapy for cystic fibrosis. Cystic Fibrosis Foundation. (n.d.). https://www.cff.org/research-clinical-trials/gene-therapy-cystic-fibrosis 

UTAustin. (n.d.). New gene-editing Tech holds promise for treating complex genetic diseases. EurekAlert! https://www.eurekalert.org/news-releases/1102990