Up to 7% of the population suffer from a rare disease, many of which are life-changing and have poor treatment options. Genome-editing technology is increasing scientists’ understanding of basic biology and human disease, allowing them to not only read genomes but to disrupt and rewrite them. The surge of research interest in this field is expected to lead to greater advances in the technology in the coming years.
The Academy of Medical Sciences had brought Dr John Leonard over from the USA to speak as the Keynote at the Academy’s FORUM* annual lecture, entitled ‘Transforming Medicine: The Promise of Human Genome Editing for Rare and Genetic Disease’. Dr Leonard is CEO of Intellia Therapeutics, one of the companies to come out of the labs that developed the CRISPR-cas9 technology. His biography can be found here.
Dr Leonard was at the SMC in advance of the Academy lecture to give his thoughts on the potential of CRISPR to treat rare genetic diseases and address questions including:
* The Academy’s FORUM, established in 2003, provides an independent platform where researchers, research funders and research users from across academia, industry, government, NHS and the charity, regulatory and wider healthcare sectors are brought together. The Academy’s FORUM annual lecture recognises a key individual working at the interface of academia, industry and healthcare.