Engineering structural variants to interrogate genome function

J Koeppel, J Weller, T Vanderstichele, L Parts - Nature Genetics, 2024 - nature.com
Structural variation, such as deletions, duplications, inversions and complex
rearrangements, can have profound effects on gene expression, genome stability …

Leveraging CRISPR gene editing technology to optimize the efficacy, safety and accessibility of CAR T-cell therapy

T Lei, Y Wang, Y Zhang, Y Yang, J Cao, J Huang… - Leukemia, 2024 - nature.com
Abstract Chimeric Antigen Receptor (CAR)-T-cell therapy has revolutionized cancer immune
therapy. However, challenges remain including increasing efficacy, reducing adverse events …

Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells

AA Sousa, C Hemez, L Lei, S Traore… - Nature biomedical …, 2024 - nature.com
Prime editing (PE) enables precise and versatile genome editing without requiring double-
stranded DNA breaks. Here we describe the systematic optimization of PE systems to …

Removal of TREX1 activity enhances CRISPR–Cas9-mediated homologous recombination

ME Karasu, E Toufektchan, Y Chen, A Albertelli… - Nature …, 2024 - nature.com
Abstract CRISPR–Cas9-mediated homology-directed repair (HDR) can introduce desired
mutations at targeted genomic sites, but achieving high efficiencies is a major hurdle in …

A benchmarked, high-efficiency prime editing platform for multiplexed dropout screening

A Cirincione, D Simpson, W Yan, R McNulty… - Nature …, 2024 - nature.com
Prime editing installs precise edits into the genome with minimal unwanted byproducts, but
low and variable editing efficiencies have complicated application of the approach to high …

Multimodal scanning of genetic variants with base and prime editing

O Belli, K Karava, R Farouni, RJ Platt - Nature Biotechnology, 2024 - nature.com
Mutational scanning connects genetic variants to phenotype, enabling the interrogation of
protein functions, interactions and variant pathogenicity. However, current methodologies …

Next-generation CRISPR technology for genome, epigenome and mitochondrial editing

CH Lau, QL Liang, H Zhu - Transgenic Research, 2024 - Springer
The application of rapidly growing CRISPR toolboxes and methods has great potential to
transform biomedical research. Here, we provide a snapshot of up-to-date CRISPR …

Prime editing: Therapeutic advances and mechanistic insights

JB Murray, PT Harrison, J Scholefield - Gene Therapy, 2024 - nature.com
We are often confronted with a simple question,“which gene editing technique is the best?”;
the simple answer is “there isn't one”. In 2021, a year after prime editing first made its mark …

Precise kilobase-scale genomic insertions in mammalian cells using PASTE

CW Fell, C Schmitt-Ulms, DV Tagliaferri… - Nature …, 2024 - nature.com
Programmable gene integration technologies are an emerging modality with exciting
applications in both basic research and therapeutic development. Programmable addition …

Open-ended molecular recording of sequential cellular events into DNA

TB Loveless, CK Carlson, CA Dentzel Helmy… - Nature Chemical …, 2024 - nature.com
Genetically encoded DNA recorders noninvasively convert transient biological events into
durable mutations in a cell's genome, allowing for the later reconstruction of cellular …