A gene-editing protein, small enough to fit into adeno-associated viruses (AAVs) that infect humans has been identified. As reported by Precision Medicine, these compact AAVs can be modified so that they efficiently transport targeted therapy to the cells.
However, the discovery presents a new urgency. There is now a need for small EbCas12a so that they can be combined into one crRNA and then into one AAV. After conducting experiments, the researchers found the EbCas12a protein demonstrated DN cleavage outside the body (in vitro). A point mutation, defined as altered DNA sequence, was then added by researchers creating a variant to EbCas12a. PCSK9 was targeted by using AAV-enEbCas12a in mice as it reduces the cholesterol levels in the blood.
The Result
The injection created a significant decrease in the serum cholesterol of the six mice that had received AAV-enEbCas12a. On the contrary, there was no improvement in the other six mice that had received phosphate-buffered saline. Bacteria go on the defensive against viruses with the use of CRISPR, the genetic tool used as scissors to cut the genetic code.
The result is a DNA template that repairs the defective gene. Professor Wang stated that enEbCas12a is a potential tool for genome editing as well as being a platform for AAV gene therapy.
