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ASH 2025 | The potential to “reset” disease progression in MPNs through genome editing
In this video, Roman Doll, BSc, MSc, University of Oxford, Oxford, UK, briefly discusses the potential of using genome editing in JAK2 V617F-driven myeloproliferative neoplasms (MPNs) to reduce the allele burden and “reset” disease progression in a way that is not achieved by current JAK2 inhibitors. This interview took place at the 67th ASH Annual Meeting and Exposition, held in Orlando, FL.
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Transcript
So far what we’re doing is very much a highly experimental approach. We have undertaken experiments in cellular models, we’ve undertaken some mouse work, but there’s definitely a lot of steps that need to be taken if this can ever be translated into a clinical strategy. If, however, this translation would be successful, the way in which this would be different is that we’re sort of targeting the root cause on the genetic level...
So far what we’re doing is very much a highly experimental approach. We have undertaken experiments in cellular models, we’ve undertaken some mouse work, but there’s definitely a lot of steps that need to be taken if this can ever be translated into a clinical strategy. If, however, this translation would be successful, the way in which this would be different is that we’re sort of targeting the root cause on the genetic level. And we sort of think about this as a sort of reset, a temporal reset of the disease. If we are able to genetically inactivate, say, 95% of mutant copies of JAK2, we’re sort of resetting disease progression by many decades. Since JAK2-driven MPNs progress very slowly, there are often years or decades from acquisition of the mutation to actual clinical manifestation of the disease. If we can genetically reduce the allele burden, we are actually sort of turning back the clocks in the patient in a way that probably is not achieved, at least by current JAK2 inhibitors.
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