A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher.
Tag: CRISPRs
Find-and-replace genome editing with CRISPR: A promising therapeutic Strategy
CRISPR-Cas9-based genome editing offers hope for treating severe immunodeficiency disorders like SCID, with a novel approach for precise gene replacement, reducing risks and potentially aiding various genetic diseases.
‘Glow-in-the-dark’ proteins could help diagnose viral diseases
Many highly sensitive diagnostic tests for viral diseases still require complicated techniques. But now, a team reporting in ACS Central Science has developed a sensitive method that analyzes viral nucleic acids quickly and can be completed in one step with “glow-in-the-dark” proteins.
Novel organoid models to study non-alcoholic fatty liver disease
Researchers from the Organoid group (former Clevers group, Hubrecht Institute) together with researchers from the Princess Máxima Center for pediatric oncology established novel human organoid models of fatty liver disease.