29 January 2018
ByAppeared in BioNews 935
The US National Institutes of Health (NIH) has announced a plan to grant US$190 million for genome editing research. The new programme, Somatic Cell Genome Editing, which is supported by the NIH Common Fund will award funds to biomedical researchers over the next six years.
'Genome editing technologies such as CRISPR/Cas9 are revolutionising biomedical research,' said Dr Francis Collins, director of NIH in Bethesda, Maryland. 'The focus of the Somatic Cell Genome Editing programme is to dramatically accelerate the translation of these technologies to the clinic for treatment of as many genetic diseases as possible.'
Genome editing techniques such as CRISPR/Cas9 present the opportunity to potentially treat disease by removing the DNA at fault from a cell, essentially 'editing' it out.
In the first public discussion of the programme on 1 September 2017, Dr Mary Ellen Perry, a programme leader in the agency's Office of Strategic Coordination said: 'We're hoping that we've come up with a concept that would help to accelerate the approaches for therapeutic genome editing. There are thousands of incurable genetic diseases which now at least potentially or theoretically are treatable through these new gene editing technologies.'
The focus of the programme, is of genome editing techniques in somatic cells - non-reproductive body cells. Successful therapeutics in somatic cells would not be inherited and so does not raise the ethical concerns associated with permanent germline genome editing.
The purpose of the programme is to assemble a genome editing 'toolkit' of knowledge, methods, and tools to be shared with the scientific community. More specifically it aims to improve the delivery mechanisms for targeted genome editing tools, develop new and improved genome editors, and design assays that can be used to evaluate the safety and efficacy of those tools.
Although much excitement exists around the advances made in genome editing and their potential clinical benefits, concerns have been raised by recent research at Stanford University that suggested that CRISPR treatment could trigger an immune response rendering therapy at best ineffective, but possibly dangerous (see BioNews 933). The NIH's financial support for the programme itself is also conditional upon annual budget appropriations.Scientists are positive about the programme. As Dr Perry said at the presentation of concept: 'What comes out of this programme, could impact everything from common disorders to rare diseases. It's almost difficult to think of a condition that this might not be applicable to.'