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Genetic editing of individual cells points to late targets for developmental disorders

A new gene editing method uses the CRISPR technique to modify the cells of an organ in vivo, creating a mosaic used to identify the effects of each altered gene. Scientists from the Swiss Federal Institute of Technology (ETH) in Zürich developed this technology called AAV-Perturb-seq, based on adeno-associated virus (AAV) to target, edit and analyze single-cell genetic perturbations.
Swiss Federal Institute Of Technology Swiss Federal Institute 22q11 2 Deletion Syndrome Adeno Associated Viruses Gene Editing Eth Zurich

Arrayed CRISPR Screening for Revolutionizing Target Discovery

In this interview conducted at SLAS EU 2023 in Brussels, Belgium, we spoke to Ulrike Kuenzel, Associate Director in the Functional Genomics department at AstraZeneca about the application of arrayed CRISPR screening for target identification and more.
United Kingdom Bruxelles Capitale Cancer Research Horizon Genomics Center University Of Oxford Society For Laboratory Automation

CRISPR-Cas9 promoting fetal hemoglobin production improves severe sickle cell disease

1. In this randomized controlled trial, infusion of OTQ923 disrupted inhibitory HBG1 and HBG2 promoter regions of the gamma-globin gene. 2. This disruption resulted in sustained production of fetal hemoglobin and improved symptoms of severe sickle cell disease (SCD). Evidence Rating Level: 2 (Good) Study Rundown: SCD is caused by mutations disrupting the function of
Minute Medicine Inc Rating Level Chronic Disease Crispr Cas9 Fetal Hemoglobin Sickle Cell Disease

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