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Article: P.F508del editing in cells from cystic fibrosis patients - Coggle…
Article: P.F508del editing in cells from cystic fibrosis patients
Problem
Caused by mutation in the CFTR gene
Most common mutation is p.F508del
Goal of Study
Use CRISPR/ Cas9 gene editing
To correct the p.F508del mutation
How they did it
CRISPR/ Cas9 enzyme
CF patient cells (cell line and iPSCs)
DNA template to fix the mutation
What happened after cutting DNA
NHEJ
Quick repair
Not precise it makes small error
HDR
Uses DNA template
Precisely fixes mutation
Results
Some mutation correction happened
HDR was low efficiency
Editing worked better in some cell types than others
Conclusion
CRISPR can correct the mutation
Efficiency needs improvement before clinical use