Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

AV Anzalone, LW Koblan, DR Liu - Nature biotechnology, 2020 - nature.com
The development of new CRISPR–Cas genome editing tools continues to drive major
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …

CRISPR in cancer biology and therapy

A Katti, BJ Diaz, CM Caragine, NE Sanjana… - Nature Reviews …, 2022 - nature.com
Over the past decade, CRISPR has become as much a verb as it is an acronym,
transforming biomedical research and providing entirely new approaches for dissecting all …

[HTML][HTML] A next generation connectivity map: L1000 platform and the first 1,000,000 profiles

A Subramanian, R Narayan, SM Corsello, DD Peck… - Cell, 2017 - cell.com
We previously piloted the concept of a Connectivity Map (CMap), whereby genes, drugs,
and disease states are connected by virtue of common gene-expression signatures. Here …

Autism genes converge on asynchronous development of shared neuron classes

B Paulsen, S Velasco, AJ Kedaigle, M Pigoni… - Nature, 2022 - nature.com
Genetic risk for autism spectrum disorder (ASD) is associated with hundreds of genes
spanning a wide range of biological functions,,,,–. The alterations in the human brain …

Evolved Cas9 variants with broad PAM compatibility and high DNA specificity

JH Hu, SM Miller, MH Geurts, W Tang, L Chen, N Sun… - Nature, 2018 - nature.com
A key limitation of the use of the CRISPR–Cas9 system for genome editing and other
applications is the requirement that a protospacer adjacent motif (PAM) be present at the …

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

BP Kleinstiver, V Pattanayak, MS Prew, SQ Tsai… - Nature, 2016 - nature.com
CRISPR–Cas9 nucleases are widely used for genome editing but can induce unwanted off-
target mutations. Existing strategies for reducing genome-wide off-target effects of the widely …

Genome-wide CRISPR screens reveal host factors critical for SARS-CoV-2 infection

J Wei, MM Alfajaro, PC DeWeirdt, RE Hanna… - Cell, 2021 - cell.com
Identification of host genes essential for severe acute respiratory syndrome coronavirus 2
(SARS-CoV-2) infection may reveal novel therapeutic targets and inform our understanding …

Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9

JG Doench, N Fusi, M Sullender, M Hegde… - Nature …, 2016 - nature.com
CRISPR-Cas9–based genetic screens are a powerful new tool in biology. By simply altering
the sequence of the single-guide RNA (sgRNA), one can reprogram Cas9 to target different …

Evaluation of off-target and on-target scoring algorithms and integration into the guide RNA selection tool CRISPOR

M Haeussler, K Schönig, H Eckert, A Eschstruth… - Genome biology, 2016 - Springer
Background The success of the CRISPR/Cas9 genome editing technique depends on the
choice of the guide RNA sequence, which is facilitated by various websites. Despite the …

Perturb-Seq: dissecting molecular circuits with scalable single-cell RNA profiling of pooled genetic screens

A Dixit, O Parnas, B Li, J Chen, CP Fulco… - cell, 2016 - cell.com
Genetic screens help infer gene function in mammalian cells, but it has remained difficult to
assay complex phenotypes—such as transcriptional profiles—at scale. Here, we develop …