The 2 Minute Medicine Podcast Episode 29
Welcome to the 2 Minute Medicine Podcast, summarizing the latest medical studies, curated and written by practicing physicians. On this ...
Tag: CRISPR
![CRISPR-mediated gene activation rescues obesity caused by haploinsufficiency [PreClinical]](https://www.2minutemedicine.com/wp-content/uploads/2019/03/CRISPR-350x250.jpg)
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. ...
CRISPR-mediated gene activation rescues obesity caused by haploinsufficiency [PreClinical]
1. Haploinsufficiency of Sim1 or Mc4r in the hypothalamus result in an obesity phenotype in mice and humans. 2. In ...
CRISPR-Cas9 corrects hypertrophic cardiomyopathy gene mutation in human preimplantation embryos [PreClinical]
1. The gene editing tool CRISPR-Cas9 was used to correct a mutant paternal MYBPC3 allele in human preimplantation embryos. 2. ...
Genome editing using CRISPR-Cas9 could correct fatal metabolic liver disease [PreClinical]
1. Viral vectors were used to deliver the correct sequence for the ornithine transcarbamylase (OTC) gene, the guide sequence to ...
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