One of the world’s most powerful genome-editing tools CRISPR is beginning to show just how efficient it can be.
Researchers from the Perelman School of Medicine at the University of Pennsylvania have developed an innovative dual-gene-therapy approach to delivering the editing tool into mice to treat haemophilia B. The disorder is also known as Factor IX Deficiency which is caused by a defective clotting protein. In some cases that protein is missing completely.
The research team consisted of associate professor in the Penn Gene Therapy Program, Lili Wang; professor of medicine and GTP director, James M Wilson. The team also consisted of visiting scientist at the Wilson lab, Yang Yang and John White, McMenamin Deirdre, and Peter Bell, all from the University of Pennsylvania.
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Their research has been presented at the 58th Annual American Society of Hematology Meeting and Expo in San Diego earlier this week.
This pre-clinical study is proof that the concept of using CRISPR gene-targeting approach to help treat various diseases and illnesses, specifically haemophilia B. “Basically, we cured the mice,” said Wang in a press release.
CRISPR has shown even more promise in this pre-clinical trial
To help validate their research, the team knocked out the clotting factor in specific mice and begun the experiment. Using a two-vector approach the team decided that the first vector would release the SaCas9 gene. This gene used a specific liver promoter to ensure that the gene-editing tool finds its way to the liver, the site responsible for producing the clotting factor IX.
The second vector contained an RNA sequence that targets a specific part of the DNA strand of the clotting factor. This approach is more potent and accurate than most other approaches. The team then used adeno-associated viral vectors to deliver the specific components to the mouse’s liver cells.
Wang and her team administered increasing doses to newborns as well as adult mice without the clotting gene, what they observed was that the mice began to show stable factor IX activity over a four-month period.
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