#gene-therapy

#gene-therapy

The revolutionary treatment is administered in just one single dose, with no need for further doses, but it is so sophisticated that it can cost around one million euros. Eliana received it a decade ago and now leads a completely normal life. She gets great grades in school, plays basketball, and has even joined the school choir. It's incredible, her father recounts with emotion.

Research suggests that ancient viral DNA embedded in the human genome is playing a key role in early embryo development. Around 8% of our genome consists of endogenous retrovirus DNA - the remnants of ancient infections, but knowledge of their activity is limited. Now, a team shows that these sequences are required for the correct development of lab-derived embryo analogues, and for the switching on of human-specific genes.

The word skin has come to stand for superficiality and lack of depth, but human skin defies such connotations. For one thing, it is enormous, accounting for roughly 15% of a person's body weight. Skin is also remarkable for its diverse roles: protection against pathogens, temperature regulation and, of course, sensation. Like any part of the body, skin is fallible to disease. But scientists are making great strides in protecting and improving the organ's health.

"It may be one of the first dementias to have a definitive treatment, a cure if you like, a really transformative treatment that allows people to live much longer and much more normal lives..."

The transformation in medicine driven by personalized gene therapies promises cures for many diseases, including cancer, diabetes, and autoimmune conditions, but widespread application may take decades.

CRISPR offers more control than viral carriers do, but typically requires the cutting of DNA - raising the chances of unwanted mutations and incomplete repair.