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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.