"As humans, we all want the same thing, a life that's full of good experiences, more time with family, with friends, more time to love, but sometimes genetic illness can cut that short or really, for all of us at some point, our body breaks down. And our bodies are genetic machines. For many diseases, the cause of the disease is a mutation in the genome. Gene therapy is a vision that many have had for decades, more than 50 years."
"The power of genetic technology is that once you get inside of cells with a DNA molecule, that molecule can stay there for the lifetime of the cell. So it's the potential for a one-time treatment for a disease where you wouldn't otherwise be able to reach the cells and solve for the root cause of the disease. Today though, for the most part, the genome you're born with is the genome you die with."
Genetic illnesses often result from mutations in the genome. Gene therapy delivers DNA molecules into cells to correct root causes and can provide durable effects because introduced DNA can persist for the life of a cell, enabling potential one-time treatments. The primary obstacle has been efficient, tissue-specific delivery of genetic payloads into target cells at therapeutic doses. Advances in capsid engineering and machine learning aim to design viral vectors that better reach specific tissues and evade immune barriers. An AI-powered capsid engineering platform was developed from prior laboratory research to improve delivery and help make gene therapy a mainstream, curative medicine.
Read at Big Think
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