The Power and Precision of Editing

 

Science Daily explains a new technology developed at the University of Pennsylvania that significantly advances genetic engineering. Called minimal, versatile genetic perturbation technology (mvGPT), it integrates gene editing and gene expression regulation into one platform. This means it can activate, deactivate, and precisely edit genes within the same cell. The breakthrough is particularly promising for treating complex diseases, such as cancer, cardiovascular disease, and/or autoimmune diseases, that stem from multiple genetic factors because it allows for multifaceted manipulation of the genetic code in a targeted and efficient way. This could lead to more effective therapies by addressing the underlying genetic causes of diseases directly.

Currently, the Mayo Clinic and Johns Hopkins Medicine discuss many successful treatments for these diseases, such as pharmacological therapy. This involves using drugs that can influence the pathways influenced by genetic changes, also known as targeted cancer therapies, that inhibit specific proteins produced by mutated genes. There is also immunotherapy, specifically treating cancer, which uses the body’s immune system to target and destroy cells with abnormal genetic expressions. Hormonal therapy is known to be used for conditions like diabetes or thyroid disorders to regulate hormone levels affected by genetic predispositions. Lastly, lifestyle modifications for conditions such as heart disease and diabetes are when changes in diet, exercise, and other lifestyle factors can significantly impact disease progression. Overall, these therapies have improved the quality of life and survival rates for many patients, though they don’t cure the genetic root causes of the disease. Hopefully, as genetic engineering continues to improve, these diseases could be essentially cured at the molecular level.

Links:

https://www.sciencedaily.com/releases/2025/01/250108144015.htm

https://www.mayoclinicplatform.org/2023/03/29/balancing-lifestyle-medicine-and-pharmacotherapy/

https://www.hopkinsmedicine.org/news/articles/2016/01/immunotherapy-plus-short-term-hormonal-therapy-promising-results