Base editing: advances and therapeutic opportunities

EM Porto, AC Komor, IM Slaymaker… - Nature Reviews Drug …, 2020 - nature.com
Base editing—the introduction of single-nucleotide variants (SNVs) into DNA or RNA in
living cells—is one of the most recent advances in the field of genome editing. As around …

Integrating non-mammalian model organisms in the diagnosis of rare genetic diseases in humans

S Yamamoto, O Kanca, MF Wangler… - Nature Reviews …, 2024 - nature.com
Next-generation sequencing technology has rapidly accelerated the discovery of genetic
variants of interest in individuals with rare diseases. However, showing that these variants …

Lipid droplets and peroxisomes are co-regulated to drive lifespan extension in response to mono-unsaturated fatty acids

K Papsdorf, JW Miklas, A Hosseini, M Cabruja… - Nature cell …, 2023 - nature.com
Dietary mono-unsaturated fatty acids (MUFAs) are linked to longevity in several species. But
the mechanisms by which MUFAs extend lifespan remain unclear. Here we show that an …

[HTML][HTML] Individual cell types in C. elegans age differently and activate distinct cell-protective responses

AE Roux, H Yuan, K Podshivalova, D Hendrickson… - Cell Reports, 2023 - cell.com
Aging is characterized by a global decline in physiological function. However, by
constructing a complete single-cell gene expression atlas, we find that Caenorhabditis …

Applications of CRISPR technologies in research and beyond

R Barrangou, JA Doudna - Nature biotechnology, 2016 - nature.com
Programmable DNA cleavage using CRISPR–Cas9 enables efficient, site-specific genome
engineering in single cells and whole organisms. In the research arena, versatile CRISPR …

Robust Genome Editing with Short Single-Stranded and Long, Partially Single-Stranded DNA Donors in Caenorhabditis elegans

GA Dokshin, KS Ghanta, KM Piscopo, CC Mello - Genetics, 2018 - academic.oup.com
A robust genome editing pipeline is critical to the vitality of a modern genetic laboratory.
Previous studies have shown that Cas9 ribonucleoprotein (RNP)-based editing can be …

CRISPR/Cas9-based genome editing for disease modeling and therapy: challenges and opportunities for nonviral delivery

HX Wang, M Li, CM Lee, S Chakraborty… - Chemical …, 2017 - ACS Publications
Genome editing offers promising solutions to genetic disorders by editing DNA sequences
or modulating gene expression. The clustered regularly interspaced short palindromic …

Genome-editing technologies: principles and applications

T Gaj, SJ Sirk, S Shui, J Liu - Cold Spring Harbor …, 2016 - cshperspectives.cshlp.org
Targeted nucleases have provided researchers with the ability to manipulate virtually any
genomic sequence, enabling the facile creation of isogenic cell lines and animal models for …

Melting dsDNA Donor Molecules Greatly Improves Precision Genome Editing in Caenorhabditis elegans

KS Ghanta, CC Mello - Genetics, 2020 - academic.oup.com
Melting and fast cooling double stranded DNA donor molecules prior to injection
dramatically increases the frequency of homology-directed repair for edits such as insertions …

The autophagy protein ATG9A enables lipid mobilization from lipid droplets

E Mailler, CM Guardia, X Bai, M Jarnik… - Nature …, 2021 - nature.com
The multispanning membrane protein ATG9A is a scramblase that flips phospholipids
between the two membrane leaflets, thus contributing to the expansion of the phagophore …