Helping these efforts, CHOP haematologist Gerd Blobel, is mapping the transcriptional regulation of fetal haemoglobin. Rather than relying on gene editing or toxic chemotherapies to dial up the ...

Key criticisms of the article include the current inaccuracy of gene-editing technologies, the challenges in accurately identifying causal gene variants, and the possibility of pleiotropic effects ...

Techniques: CRISPR/Cas9 gene editing, lipid nanoparticle delivery ... By integrating these projects, The Barrozo Lab continues to push the boundaries of maternal-fetal medicine and environmental ...

With the addition of Casgevy, Children’s Hospital Los Angeles now offers two gene therapies for sickle cell, the first being Lyfgenia.

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Gene editing using base editing has been shown to extend the lifespan of mice with prion disease by approximately 50%. This approach involves a single-letter DNA change that reduces prion protein ...

Researchers have developed a gene-editing treatment for prion disease that extends lifespan by about 50 percent in a mouse model of the fatal neurodegenerative condition. The treatment ...

This study presents an optimized CRISPR-Cas12a system for large-scale genetic screening, revealing critical genes involved in lymphoma development and therapy.

BEAM’s pipeline of gene editing therapies is based on its proprietary ... with BEAM-101 led to a robust and durable increase in fetal hemoglobin of more than 60% and a reduction in sickle ...