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ISAL 2025 | Using CRISPR/Cas9 to identify potential therapeutic targets in acute leukemia PDX cells
Binje Vick, PhD, Helmholtz Munich, Munich, Germany, comments on the potential of combining CRISPR screens with patient-derived xenograft (PDX) models to identify new therapeutic targets in acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML). Dr Vick highlights a study that validated targets with single sgRNA and in vivo therapy trials. This approach holds promise for identifying new targets for treatment with available inhibitors. This interview took place at the 19th International Symposium on Acute Leukemias (ISAL XIX) in Munich, Germany.
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Transcript
So with the other project, this was really difficult and we’re really at the edge of what is feasible. So what we do is we express Cas9 and PDX models, which is not feasible in all the models that we have established. And we first performed CRISPR screens with customized libraries to be able to define new vulnerabilities. So new targets that we can then also treat with available inhibitors...
So with the other project, this was really difficult and we’re really at the edge of what is feasible. So what we do is we express Cas9 and PDX models, which is not feasible in all the models that we have established. And we first performed CRISPR screens with customized libraries to be able to define new vulnerabilities. So new targets that we can then also treat with available inhibitors. So the library covered genes that can also be targeted by inhibitors. And we did this in several ALL and AML PDX models and then uncovered shared but also individual vulnerabilities. And then we confirmed this by single validations with single sgRNAs and could also confirm these findings of the CRISPR screens and are currently also performing in vivo therapy trials to see if these samples then also respond in vivo towards these inhibitors. One problem here is that inhibitors are often of course not as specific as an sgRNA. So we are currently dealing with some issues on keeping this specificity. But what we see is that really the results that come up from these CRISPR screens can also be then validated by in vivo therapy and so we hope that with this approach we can really find putative also new vulnerabilities in ALL and AML.
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