Question

These questions relate to: Prime Editing

1. Describe (briefly) the scientific principle behind how the technology works. This may be complex and the goal here is to not get bogged down in so much detail that this becomes incomprehensible.

2. What was the basic research which made this possible?

3. Can you find any information from patents?

Answer 1

A new gene editing technique called Prime editing, is a precise and versatile genome editing method used to directly writes new genetic information into a specified genome site using a catalytically impaired Cas9 endonuclease fused to an engineered reverse transcriptase, programmed with a prime editing guide RNA (pegRNA) that both specifies the target site and encodes the desired edit. Once the new genetic material is incorporated into the cut strand of DNA, the prime editor nicks the unedited strand, signaling to the cell to rebuild it to match the edited strand.

It allows to rewrite DNA by only cutting a single strand to add, remove, or replace base pairs. The method allows to edit more types of genetic mutations.

Prime editing is more complex than CRISPR editing. It requires three separate steps in which the DNA must match up with parts of the prime editing system. Prime editing requires this first step, but also includes two more components, a part of the guide RNA called the primer must also bind to the target site and the newly introduced DNA must bind to the original site. If any one of those three DNA pairing events fail, then prime editing can’t proceed and these three independent pairing events each provide an opportunity to reject off-target sequences.

Prime editing has been used to target genes underlying Tay-Sachs disease and sickle cell anemia. The mutations were changed back to healthy DNA sequences with 35–55 percent efficiency.

The Technology have been patented by Broad Institute and licenced to Prime Medicine, under the institute’s “inclusive innovation” model,