In vivo prime editing of a metabolic liver disease in mice

D Böck, T Rothgangl, L Villiger, L Schmidheini… - Science translational …, 2022 - science.org
Prime editing is a highly versatile CRISPR-based genome editing technology that works
without DNA double-strand break formation. Despite rapid technological advances, in vivo …

Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

L Villiger, HM Grisch-Chan, H Lindsay, F Ringnalda… - Nature medicine, 2018 - nature.com
CRISPR–Cas-based genome editing holds great promise for targeting genetic disorders,
including inborn errors of hepatocyte metabolism. Precise correction of disease-causing …

[HTML][HTML] WT-PE: Prime editing with nuclease wild-type Cas9 enables versatile large-scale genome editing

R Tao, Y Wang, Y Hu, Y Jiao, L Zhou, L Jiang… - Signal transduction and …, 2022 - nature.com
Large scale genomic aberrations including duplication, deletion, translocation, and other
structural changes are the cause of a subtype of hereditary genetic disorders and contribute …

[HTML][HTML] Prime editing: genome editing for rare genetic diseases without double-strand breaks or donor DNA

IG Matsoukas - Frontiers in Genetics, 2020 - frontiersin.org
An article published in Nature (Anzalone et al., 2019) reports the development of a genome
editing experimental approach that mediates all possible base-to-base conversions,“indels,” …

A split prime editor with untethered reverse transcriptase and circular RNA template

B Liu, X Dong, H Cheng, C Zheng, Z Chen… - Nature …, 2022 - nature.com
Delivery and optimization of prime editors (PEs) have been hampered by their large size
and complexity. Although split versions of genome-editing tools can reduce construct size …

[HTML][HTML] Prime editing for human gene therapy: where are we now?

K Godbout, JP Tremblay - Cells, 2023 - mdpi.com
Gene therapy holds tremendous potential in the treatment of inherited diseases. Unlike
traditional medicines, which only treat the symptoms, gene therapy has the potential to cure …

[HTML][HTML] Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice

P Liu, SQ Liang, C Zheng, E Mintzer, YG Zhao… - Nature …, 2021 - nature.com
Prime editors (PEs) mediate genome modification without utilizing double-stranded DNA
breaks or exogenous donor DNA as a template. PEs facilitate nucleotide substitutions or …

[HTML][HTML] Prime editing: advances and therapeutic applications

Z Zhao, P Shang, P Mohanraju, N Geijsen - Trends in Biotechnology, 2023 - cell.com
Clustered regularly interspaced short palindromic repeats-associated protein 9 (CRISPR–
Cas)-mediated genome editing has revolutionized biomedical research and will likely …

[HTML][HTML] Evaluating CRISPR-based prime editing for cancer modeling and CFTR repair in organoids

MH Geurts, E de Poel… - Life science …, 2021 - life-science-alliance.org
Prime editing is a recently reported genome editing tool using a nickase-cas9 fused to a
reverse transcriptase that directly synthesizes the desired edit at the target site. Here, we …

A prime editor mouse to model a broad spectrum of somatic mutations in vivo

ZA Ely, N Mathey-Andrews, S Naranjo, SI Gould… - Nature …, 2024 - nature.com
Genetically engineered mouse models only capture a small fraction of the genetic lesions
that drive human cancer. Current CRISPR–Cas9 models can expand this fraction but are …