β-Thalassemia is caused in part by a mutated β-globin gene, so researchers developed a way to replace the entire mutated gene with a healthy version...

In this brief roundup, we look at what's going on in the CRISPR Medicine field for Leber congenital amaurosis, an inherited group of rare retinal...

This week’s clinical update looks at two clinical trials for beta thalassemia using CRISPR-Cas9-edited donor-derived haematopoeitic stem cells (HSCs).

The group of Mario Amendola has used CRISPR-based editing of haematopoietic stem cells to generate a common platform for treating several rare genetic...

CRISPR-Cas is seen as the holy grail in medicine for rare and incurable genetic diseases. In this piece, we take a look at what CRISPR can do for...