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 …

Enrichment strategies to enhance genome editing

NS Mikkelsen, RO Bak - Journal of Biomedical Science, 2023 - Springer
Genome editing technologies hold great promise for numerous applications including the
understanding of cellular and disease mechanisms and the development of gene and …

Genetics of cystogenesis in base-edited human organoids reveal therapeutic strategies for polycystic kidney disease

CE Vishy, C Thomas, T Vincent, DK Crawford… - Cell Stem Cell, 2024 - cell.com
In polycystic kidney disease (PKD), microscopic tubules expand into macroscopic cysts.
Among the world's most common genetic disorders, PKD is inherited via heterozygous loss …

Development of multiplexed orthogonal base editor (MOBE) systems

QT Cowan, S Gu, W Gu, BL Ranzau… - Nature …, 2024 - nature.com
Base editors (BEs) enable efficient, programmable installation of point mutations while
avoiding the use of double-strand breaks. Simultaneous application of two or more different …

Modeling long QT syndrome type 2 on-a-chip via in-depth assessment of isogenic gene-edited 3D cardiac tissues

J Veldhuizen, HF Mann, N Karamanova… - Science …, 2022 - science.org
Long QT syndrome (LQTS) is a cardiovascular disease characterized by QT interval
prolongation that can lead to sudden cardiac death. Many mutations with heterogeneous …

Mutation-specific reporter for optimization and enrichment of prime editing

IF Schene, IP Joore, JHL Baijens, R Stevelink… - Nature …, 2022 - nature.com
Prime editing is a versatile genome-editing technique that shows great promise for the
generation and repair of patient mutations. However, some genomic sites are difficult to edit …

PEAR, a flexible fluorescent reporter for the identification and enrichment of successfully prime edited cells

DA Simon, A Tálas, PI Kulcsár, Z Biczók, SL Krausz… - Elife, 2022 - elifesciences.org
Prime editing is a recently developed CRISPR/Cas9 based gene engineering tool that
allows the introduction of short insertions, deletions, and substitutions into the genome …

Universal toxin-based selection for precise genome engineering in human cells

S Li, N Akrap, S Cerboni, MJ Porritt… - Nature …, 2021 - nature.com
Prokaryotic restriction enzymes, recombinases and Cas proteins are powerful DNA
engineering and genome editing tools. However, in many primary cell types, the efficiency of …

Manipulating and studying gene function in human pluripotent stem cell models

E Balmas, F Sozza, S Bottini, ML Ratto, G Savore… - FEBS …, 2023 - Wiley Online Library
Human pluripotent stem cells (hPSCs) are uniquely suited to study human development and
disease and promise to revolutionize regenerative medicine. These applications rely on …

Genome editing for crop improvement: A perspective from India

A Bhattacharya, V Parkhi, B Char - In Vitro Cellular & Developmental …, 2021 - Springer
Human population is expected to reach to about 10 billion by 2050. Climate change affects
crop production, thus posing food security challenges. Conventional breeding alone will not …