You had questions for David Liu about CRISPR, prime editing, and advice to young scientists. He has answers
While Mother Nature takes first prize in the race to develop new forms of CRISPR, biochemist David Liu is a close runner-up — and his CRISPR inventions have the potential to treat or prevent a long list of dreaded diseases, from progeria to Tay-Sachs. In 2016 Liu and his junior colleagues invented CRISPR “base editing,” which seamlessly changes a single DNA letter; that simplest of all edits may be all that’s required to repair mutations that cause thousands of inherited diseases. Last month he gave the world “prime editing,” which can delete long lengths of disease-causing DNA or insert DNA to repair dangerous mutations, all without triggering the chaotic (and possibly harmful) genome responses introduced by other forms of CRISPR.
In his spare time, Liu co-founded Editas Medicine, Beam Therapeutics, and Prime Medicine. STAT invited readers to submit questions to Liu on the new technology. He also received some questions directly. From his perch at the Broad Institute of MIT and Harvard, Liu answered:
Anonymous: I saw the announcement regarding the deal between Prime Medicine & Beam Therapeutics. Will Prime Medicine develop its own therapeutic applications of prime editing?
As was announced, prime editing for human therapeutics will be jointly developed by both Prime Medicine and Beam Therapeutics, each focusing on different types of edits and distinct disease targets, which will help avoid redundancy and allow us to cover more disease territory overall. The companies will also share knowledge in prime editing as well as in accompanying technologies, such as delivery and manufacturing.
Anonymous: Why do another startup instead of putting the technology into Beam, where you’re a co-founder?
I believe patients will be best served by having multiple teams of people dedicated to transitioning each new technology into human therapeutics. The relationship between Beam Therapeutics and Prime Medicine was designed to maximize patients’ interests, so that as many patients as possible can benefit from base editing or prime editing technology. This cooperative arrangement also minimizes the unnecessary expenditure of time and resources on critical technologies such as delivery and manufacturing that are needed to bring any editing technology to patients. It also ensures that when base editing and prime editing both offer potential solutions to editing a disease target, both technologies can be explored non-competitively to maximize patient benefit.
Zoe A.: Can you please compare the pros and cons of prime editing versus base editing?
The first difference between base editing and prime editing is that base editing has been widely used for the past 3 1/2 years in organisms ranging from bacteria to plants to mice to primates. Addgene tells me that the DNA blueprints for base editors from our laboratory have been distributed more than 7,500 times to more than 1,000 researchers around the
