Gene therapy has the potential to dramatically benefit patients with genetic blood disorders, but a small study on participants in a clinical trial for sickle cell disease suggests that a key process ...

With a shared purpose, we can realize the true promise of CRISPR and improve healthcare, providing hope to more patients with ...

Alternative splicing is a genetic process where different segments of genes are removed, and the remaining pieces are joined together during transcription to messenger RNA (mRNA). This mechanism ...

Polyplus, part of Sartorius, agreed to collaborate with MarkHerz, a Korean cell and gene therapy (CGT) CDMO, to develop new CGT efficiency standards focused on decreasing cost and increasing quality ...

Genetic testing can confirm a muscular dystrophy (MD) diagnosis when symptoms and other tests already suggest MD. It also identifies specific gene mutations that can guide targeted treatment. Genetic ...

With the FDA expecting to approve 10–20 Cell and Gene Therapies (CGTs) annually by 2025, it is evident that the field will continue to hold immense commercial and medical opportunities. To harness ...

Thinking of gene loss as an evolution force is a counterintuitive idea, for it is easier to think that only when we gain something -genes in this case- can we evolve. However the new work by these ...