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Publication in Cell Reports Medicine by GET-IN partners Carlon and Cereseto

GET-IN partners Marianne Carlon and Ana



GET-IN partners Marianne Carlon and Ana Cereseto recently published their work "Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells" in Cell Reports Medicine. An excellent example how organoids can be elegantly used to evaluate efficacy of gene therapy in human tissue derived in vitro models.



Some Highlights:

  • Prime editing can be used to precisely correct cystic fibrosis-causing mutations

  • Efficient functional correction in patient-derived organoids and airway epithelium

  • Machine learning tool DETECTOR functionally screens genetic strategies in primary organoids

  • Genome-wide evaluation of prime editing for L227R and N1303K confirms high fidelity


The paper received considerable interest in lay media:

 

 

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