How one lab’s decadeslong search for the ‘holy grail’ of sickle cell led to the first CRISPR therapy

In a 2008 is­sue of Sci­ence, just a page apart, were the two cru­cial dis­cov­er­ies that would form the cure for sick­le cell dis­ease.

Stu­art Orkin, a hema­tol­o­gist at Boston Chil­dren’s Hos­pi­tal, had pub­lished work iden­ti­fy­ing the ge­net­ic switch that could turn on back­up copies of life-sus­tain­ing he­mo­glo­bin pro­teins in sick­le cell pa­tients. And in the very next ar­ti­cle, a pair of mi­cro­bi­ol­o­gists from Illi­nois de­scribed a sys­tem called CRISPR — the tool that could flip that switch, but a mere cu­rios­i­ty at the time.

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