Robust and permanent rescue of endogenous CFTR function with AsCas12a in patient cells


Very significant milestone published today. No off target effects and highly efficient and permanent correction of cystic fibrosis causing intronic mutations with AsCas12a (a CRISPR variant) which paves the way for a cure for that lethal disease.

Incidentally, I was quite inspired by the thoughts of Claire Wineland, a famous CF sufferer and blogger who achieved great support for her patient community before her death last year. She emphasised that, for her personally, she had no interest in a bucket list comprised of things that would bring her a transient pleasure, but hoped to make a positive contribution and a difference in the world she could be proud of regardless of personal circumstance. Ultimately, this was how she achieved a fundamental self-respect for the person she was.