This week saw a major development for the field of gene-editing with a European pharmaceutical company applying to run the first CRISPR clinical trial.
CRISPR Therapeutics has applied to the European regulatory authorities to begin an industry-sponsored clinical human trial with a CRISPR therapy in 2018.
The CRISPR treatment will be designed for patients with sickle cell disease and β-thalassemia.
I think it’s a momentous occasion for us, but also for the field in general. Just three years ago we were talking about CRISPR-based treatments as sci-fi fantasy, but here we are. Samarth Kulkarni, CEO of CRISPR Therapeutics – in Wired
If successful the technique could radically imporve the quality of life for those patients with sickle cell disease or β-thalassemia – both have specific genetic mutations that adversely affect a subunit of haemoglobin which effects how the well the bodies blood cells can carry oxygen. Symptoms range from fatigue, jaundice and severe pain to death.
How does CRISPR Work
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats.
This new gene-editing technique is based on bacterias defence system – in its most basic terms, it is a “programmable” system which can target and edits specific pieces of DNA.
Roll on 2018.