Researchers have developed a novel and more powerful form of CRISPR that offers the possibility to correct most disease-causing genetic variations in DNA – new hope for the treatment of age-related disorders?
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) and CRISPR-associated (Cas) were discovered as components of bacterial adaptive immunity in the ‘80s; however, their potential applications have not been made clear until last decade.
CRISPR-Cas9 is currently the most widely used genome editing method. It is an excellent and reliable tool to make targeted changes to the genome of living cells, opening a new era in genetic engineering and revolutionising the way we approach the treatment of numerous diseases, including age-related disorders [1].
As we described in a previous article at Longevity.Technology, research led by David Liu at the Broad Institute of MIT and Harvard has led to the recent development of a new more powerful form of this technology (which they called “prime editing”) that offers the possibility of efficient and reliable introduction of precise alterations in the gene without causing double-strand breaks in the DNA double helix [2].
By increasing the range of genomic changes that can be introduced, this technology has expanded drastically the number of diseases that can be treated using genome editing, offering the potential to correct 89% of known disease-causing genetic variations in DNA. Prime editors also make genome editing in non-dividing cells such as neurons and muscle cells, possible. Thus, the treatment of neurodegenerative or muscle disorders – which are often associated with aging – has become possible [2].
The inventors have applied for a patent securing the exclusive use of this technology for the development of human therapeutics by Prime Medicine – a company co-founded by Liu. However, the non-commercial use of prime editing is freely available to academic and non-profit researchers [3].
Companies, including Editas Medicine, Shape Therapeutics, and CRISPR Therapeutics, are already testing the potential use of first-generation CRISPR technologies for the treatment of certain diseases, such as sickle cell anaemia [3].
However, age-related disorders and other more complex diseases would be almost impossible to cure with these generation 1 approaches; prime editing and Prime Medicine now provide new levels of opportunity for discovery and potential licensing opportunities for Liu’s Prime Medicine.
[1] https://ghr.nlm.nih.gov/primer/genomicresearch/genomeediting
[2] https://www.nature.com/articles/s41586-019-1711-4
[3] http://bit.ly/2Z864Lc
