Kate
Lee
Optimizing Prime Editing Strategies for Cystic Fibrosis
Abstract profile. Full document pending author claim.
Authors:
Kate Lee, Reese Caldwell, David Liu
Date Created:
2025-01-01
Course Title:
Professor:
Not specified
About Paper:
Prime editing (PE) is a CRISPR-based genome editing tool fibrosis (CF) transmembrane conductance regulator (CFTR) that can be used to precisely correct disease-causing mutations. F508delmutation,athree-nucleotidedeletionfoundin~70%ofCF However, sequence-dependentchallengespersistinachievinghigh patients. While previously-optimized PE strategies yield moderate editing efficiencies with minimal undesired byproducts. These editing efficiencies (~20-40%), they suffer from high indel rates. byproducts include insertion-deletion mutations (indels), which We are currently optimizing PE strategies which are expected to at high frequencies can compromise gene integrity and clinical offer a significant improvement in both precision and efficacy of applicability. Clinically-viable prime editing approaches must correcting CFTR F508del. This work advances the development be optimized to reduce indel formation without compromising of clinically translatable prime editing systems with therapeutic efficiency. Here, I describe one such optimization that potential for cystic fibrosis and other genetic disorders. leverages unpublished advancements to correct the human cystic
Abstract:
Prime editing (PE) is a CRISPR-based genome editing tool fibrosis (CF) transmembrane conductance regulator (CFTR) that can be used to precisely correct disease-causing mutations. F508delmutation,athree-nucleotidedeletionfoundin~70%ofCF However, sequence-dependentchallengespersistinachievinghigh patients. While previously-optimized PE strategies yield moderate editing efficiencies with minimal undesired byproducts. These editing efficiencies (~20-40%), they suffer from high indel rates. byproducts include insertion-deletion mutations (indels), which We are currently optimizing PE strategies which are expected to at high frequencies can compromise gene integrity and clinical offer a significant improvement in both precision and efficacy of applicability. Clinically-viable prime editing approaches must correcting CFTR F508del. This work advances the development be optimized to reduce indel formation without compromising of clinically translatable prime editing systems with therapeutic efficiency. Here, I describe one such optimization that potential for cystic fibrosis and other genetic disorders. leverages unpublished advancements to correct the human cystic
Source:
Harvard / William Le, Marina Barba, Vlad Denic / 2025
Topics:
editing, prime, strategy, cystic, fibrosi, efficiency, indel, optimizing, cftr, correct, mutation, byproduct
