Gene editing

Research deepens on using "jumping genes" in CRISPR therapy

Illustration: Rebecca Zisser/Axios

Two prominent teams of scientists recently announced transposons — or "jumping genes" — can improve the precision of CRISPR gene editing.

Why it matters: While this research is still in early stages, as both teams tested their techniques on bacterial cells, experts say the technique could allow edited genes to be more precisely inserted into genomes, possibly addressing concerns with current CRISPR systems that can lead to off-target editing and random deletions or even cancer.

The most expensive drug in the world

The FDA on Friday approved Zolgensma, a gene replacement therapy from Novartis that treats spinal muscular atrophy, for use in children younger than 2.

Why it matters: The treatment attacks a debilitating genetic disease that often kills infants, and it will come with a price tag of more than $2.1 million, making Zolgensma the most expensive drug on the planet.