The elegant parallel of using CRISPR to understand disease mechanisms
CRISPR is the buzzword of the moment in the drug discovery industry – mainly due to its potential to correct disease-causing mutations. However, those using the technology need to be mindful that it is used responsibly, and possible risks are considered before use. Mark Behlke discusses the potential of CRISPR in R&D and the challenges that it presents for researchers.
CRISPR TECHNOLOGY has generated much excitement in the drug discovery realm for its ability to make precise, permanent changes to DNA in animals, as first demonstrated approximately six years ago. It is currently being evaluated in early phase clinical trials for several disorders. Diseases caused by a single gene mutation – sickle cell disease (SCD), Huntington’s disease and cystic fibrosis – are all prime targets for using CRISPR gene therapy to correct the disease-causing DNA mutations. CRISPR is also being investigated as a treatment for acquired immune deficiency syndrome (AIDS) and to improve anti-tumour immunotherapy.
While news about potential CRISPR therapeutics and the start of new clinical trials dominate headlines in the lay press, CRISPR has also become a leading research tool to help scientists better understand gene function and establish model systems of human diseases needed to translate basic scientific knowledge into new medical treatments.