Duke University Medical Center-led research has identified a human-specific DNA enhancer that regulates neural progenitor ...

Gene-editing tools such as CRISPR/Cas9 can be used to create isogenic cell lines, which can be further used to model a specific patient population. An isogenic cell line was created to model ...

These investigations utilize a combination of cell culture and genetic models including transgenic and knock out mouse lines for RNA binding proteins, CRISPR-derived mouse lines in which specific ...

James Marsh, Max Planck Institute for Biology “We really need to work out a way to access the majority rather than the model organism minority ... Scientists use CRISPR systems to genetically ...

In about a third of patients, the gene for dystrophin has small deletions or single base mutations that render it nonfunctional, which makes this gene an excellent candidate for gene editing.

In new mouse models of the disease ... To fix the dysferlin mutation, Escobar uses CRISPR-Cas9, which is often described as "gene-editing scissors" and for which a Nobel Prize was awarded in ...

A big breakthrough is being hailed in the world of gene editing, with the first custom-designed treatment using CRISPR ...

Relying on CRISPR, they then took both the stem cell ... They would then have to test that work in more mouse models, and only if they succeed there try the same technique on an elephant.

CRISPR-Cas9 enzymes can be used to edit genes ... The researchers conducted proof-of-concept experiments in human cells and a mouse model of retinitis pigmentosa, finding that the bespoke enzymes ...