CRISPR-Cas9 DNA base-editing and prime-editing

A Kantor, ME McClements, RE MacLaren - International journal of …, 2020 - mdpi.com
Many genetic diseases and undesirable traits are due to base-pair alterations in genomic
DNA. Base-editing, the newest evolution of clustered regularly interspaced short palindromic …

CRISPR-based genome editing through the lens of DNA repair

TS Nambiar, L Baudrier, P Billon, A Ciccia - Molecular cell, 2022 - cell.com
Genome editing technologies operate by inducing site-specific DNA perturbations that are
resolved by cellular DNA repair pathways. Products of genome editors include DNA breaks …

In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels

T Rothgangl, MK Dennis, PJC Lin, R Oka… - Nature …, 2021 - nature.com
Most known pathogenic point mutations in humans are C• G to T• A substitutions, which can
be directly repaired by adenine base editors (ABEs). In this study, we investigated the …

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 …

Mitigation of chromosome loss in clinical CRISPR-Cas9-engineered T cells

CA Tsuchida, N Brandes, R Bueno, M Trinidad… - Cell, 2023 - cell.com
CRISPR-Cas9 genome editing has enabled advanced T cell therapies, but occasional loss
of the targeted chromosome remains a safety concern. To investigate whether Cas9-induced …

Advances in genome editing through control of DNA repair pathways

CD Yeh, CD Richardson, JE Corn - Nature cell biology, 2019 - nature.com
Eukaryotic cells deploy overlapping repair pathways to resolve DNA damage.
Advancements in genome editing take advantage of these pathways to produce permanent …

Nuclear cGAS suppresses DNA repair and promotes tumorigenesis

H Liu, H Zhang, X Wu, D Ma, J Wu, L Wang, Y Jiang… - Nature, 2018 - nature.com
Accurate repair of DNA double-stranded breaks by homologous recombination preserves
genome integrity and inhibits tumorigenesis. Cyclic GMP–AMP synthase (cGAS) is a …

CRISPR technologies for genome, epigenome and transcriptome editing

L Villiger, J Joung, L Koblan, J Weissman… - … Reviews Molecular Cell …, 2024 - nature.com
Our ability to edit genomes lags behind our capacity to sequence them, but the growing
understanding of CRISPR biology and its application to genome, epigenome and …

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 …

DNA damage: from threat to treatment

A Carusillo, C Mussolino - Cells, 2020 - mdpi.com
DNA is the source of genetic information, and preserving its integrity is essential in order to
sustain life. The genome is continuously threatened by different types of DNA lesions, such …