A CRISPR Way To Fix Faulty Genes

A CRISPR Way To Fix Faulty Genes
by Joe Palca
June 26, 2014

“It’s really powerful, it’s a really exciting development,” says Craig Mello of the University of Massachusetts Medical School.
He won the Nobel Prize in 2006 for a different technique that also lets scientists modify how genes work.
But, Mello says, this new genetic tool – known as CRISPR for clustered regularly interspersed short palindromic repeats — is more powerful, “because now you can essentially change a genome at will to almost anything you want. The sky’s the limit.”

therapies for people with genetic blood diseases like sickle cell disease and thalassemia.

original paper:
CRISPR-Cas systems for editing, regulating and targeting genomes
Nature Biotechnology  32, 347–355 (2014)
Jeffry D Sander & J Keith Joung

Americans Win Nobel for Work in Genetic Therapy
October 02, 2006