#prime-editing

#prime-editing

A single multipurpose gene-editing tool can correct several genetic conditions by restoring proteins that have been truncated by disease-causing mutations. The method might one day overcome a key stumbling block faced by gene-editing therapies: the need to design a bespoke treatment for each disease. The new approach, called PERT, combines gene editing with engineered RNA molecules that allow protein synthesis to continue even when a mutation in the DNA tells it to stop prematurely.

Therapeutic genome-editing efforts, including more than 70 clinical trials so far, have predominantly used programmable nucleases, base editors or prime editors to disrupt or correct disease-associated genes in an allele-specific manner. These approaches have proven to be effective in patients or in animal models for the treatment of disorders such as sickle-cell disease6,7, T cell leukaemia8, hypercholesterolaemia9,10, alpha-1-antitrypsin deficiency10, chronic granulomatous disease11, progeria12, spinal muscular atrophy13, prion disease14, alternating hemiplaegia of childhood15 and many other genetic diseases. Although allele-specific therapeutic genome-editing strategies offer treatments for many serious diseases with few treatment options, the breadth of the global genetic disease crisis,