🧬 Future Shock: Genes, Ghosts & Births 😱

Science

🎧English flagFrench flagGerman flagSpanish flag

Gene Therapy Triumph: A Newborn’s Remarkable Recovery
KJ Muldoon’s story is a beacon of hope, illustrating the transformative potential of personalized gene therapies. Born with a rare genetic disorder leading to dangerous ammonia accumulation, he received an experimental “base editing” treatment that, after three doses, has resulted in his complete recovery. Remarkably, he recently took his first step and celebrated his first Christmas at home, marking a profound turning point in his life and a testament to the advancements in gene therapy.

Personalized Medicine Takes a Giant Leap
The team behind KJ’s treatment is pursuing regulatory approval through a small clinical trial, aiming to make the innovative therapy more accessible. Led by David Klein, who recently cofounded Aurora Therapeutics, the strategy focuses on a single, adaptable drug designed for individual patients, potentially reducing the need for extensive clinical trials and lowering the current cost of approximately $1 million. US regulators are showing a favorable approach to this “bespoke, personalized therapy” model.

De-Extinction Efforts: Bringing Ancient Species Back to Life
Significant advancements continue within the biotechnology sector, exemplified by Colossal Biosciences’ ambitious “de-extinction” projects. The company’s success includes the creation of “woolly mice” – rodents bearing similarities to woolly mammoths – and three dire wolves, developed through targeted genetic modifications. This innovative approach, utilizing gene editing, demonstrates the growing ability to manipulate and recreate traits from ancient species.

Ethical Crossroads: Selecting Embryo Traits
The ability to screen embryos for desired traits, like height and intelligence, is rapidly developing. Companies are offering prospective parents the chance to select embryos based on these characteristics. However, this technology faces intense ethical debate, largely due to the complexity of traits like intelligence and the limited understanding of their underlying mechanisms. Concerns about a return to eugenics and the impossibility of precise predictions remain central to the discussion.

This article is AI-synthesized from public sources and may not reflect original reporting.