Publication in Cell Reports Medicine by GET-IN partners Carlon and Cereseto
- GET-IN
- May 7, 2024
- 1 min read
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: