Gene therapy—once something out of science fiction—is now being used in real hospitals to treat real people. Gene editing has become a conversation of not only treating rare diseases but also about access, fairness, and how much control we should have over our biology.
Genes are sections of DNA that act like instruction manuals telling our cells how to build proteins. Proteins perform vital function like energy use, cellular communications, immunity and cell repair. So when people say “We are what our genes make us,” it’s because these gene-coded proteins guide our growth, health, and behaviour.
Sometimes, typos appear in these instruction manuals. They are called genetic mutations. While many mutations are harmless, some affect the protein made from the mutated gene and disrupt how the cell functions. Some cause serious diseases like cystic fibrosis, muscular atrophy or certain cancers.
But if a typo in a gene can cause illness, can we fix it like we do in a text? This is what gene therapy aims to do. The field is rapidly evolving – from treating rare genetic diseases to developing personalized treatments tailored to each person’s unique combination of genetic variations, even in common diseases like diabetes. And CRISPR-Cas is currently at the forefront of these therapies.
CRISPR-Cas is a part of a bacterial immune system. Whe