Fresher and CRISPR
The promise of genome editing is tempered for genetically heterogenous diseases. Could a new approach overcome current limitations of correcting one gene per therapy, at least for rhodopsin-dependent retinitis pigmentosa?
As the basis of ‘genome editing,’ CRISPR (clustered regularly interspaced short palindromic repeats) and CRISPR-associated systems (Cas) are attracting intense interest. In CRISPR-Cas gene modification, a guide RNA (gRNA) directs nuclease to recognize and cut a matching DNA sequence (for example, an unwanted mutation). At the same time, a replacement (therapeutic) DNA sequence is provided for the DNA repair machinery to insert at the break site.
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