Gene Editing Tools Have Potential to Change Healthcare
At this moment in time the ability to edit problematic genomes to cure disease is easier than it’s ever been, and new technologies will soon make it even simpler.
This was the opinion of Ross Wilson, principal investigator at UC Berkeley’s California Institute for Quantitative Biosciences, who spoke at the HIMSS Precision Medicine Summit on Monday (12 June), reports Healthcare IT News.
It is without doubt that there have been some intriguing recent studies made with gene editing technology, but Wilson believes that CRISPR’s “hype eclipses early success stories.” He continued to explain that in ten years from now, “it will come into its own and be safe and effective.”
The process of fixing specific inheritable diseases by accessing the genome and fixing the root cause is clearly far more complex than the “cut and paste” analogies Wilson used to explain advanced precision medicine techniques to non-scientists.
However, he referenced CRISPR-Cas9 as a “readily programmable genome-editing tool” that makes the process more intuitive and usable for researchers and clinicians than ever before.
According to Wilson, there are three main hurdles to overcome:
- Efficacy: Although cutting out a gene can be done reliably, it’s still not easy or efficient to “paste” the corrected gene.
- Delivery: Researchers still lack robust and reliable technology for sending genome-editing enzymes to the cells in need of repair.
- Genetic understanding: Clinicians’ ability to cure disease remains hamstrung by an impressive but still too limited knowledge of the underlying genetic causes.
“The total scope of genome editing will be defined by our genetic understanding,” he added.
There are currently three conceivable approaches to genome-editing, in order of prevalence, explained Wilson: ex vivo, in vivo (systemic), in vivo (targeted).
The “earliest success stories will be ex vivo” – enabling clinicians to snip and edit genomes in a petri dish with a “high-efficiency that will let you do the ‘pasting’ of the effective gene that’s hard to do in the host,” he added.
In vivo delivery is the “holy grail” added Wilson. The challenge that remains is that it’s hard to distribute effectively to the correct areas of the body.
He continued, “I think we’ll be able to see genome editing applied to things that might be a bit more elective, but could have huge impact, since they’re so common.”
Despite this, Wilson stressed that we will have to wait to check how safe effective it would be. One complication for therapeutic genome editing includes the existence of such a dizzying array of genetic diversity.
He stressed how convinced he is that genome sequencing will “come to the forefront” of primary care in the near future, with these techniques more commonly identifying genetic risk factors.
Nevertheless, this brings with it big responsibilities. Clinicians will have to design sequences to minimise off target risks, something that demands whole genome analysis, and an “established gene target will have several validated “flavours” of enzyme, and the appropriate one will be administered based on the patient’s genetic profile.”