highlighting its potential as an efficient and precise tool for gene editing applications. Establishment and robust expansion of human fetal brain organoids, their characterization and adaptation ...

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 ...

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

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

Scientists are pushing for reform, arguing that Canada’s law blocks people’s right to benefit from scientific discoveries Ben Schaub “CRISPR” is a powerful gene-editing technology that is ...

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

Vertex has entered a reimbursement agreement with NHS England enabling eligible SCD patients to access Casgevy in the public health system.

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 ...

Gene editing tools like CRISPR/Cas-9 molecular scissors ... Casgevy enables CRISPR/Cas-9 to edit the BCL11A gene in stem cells to produce fetal hemoglobin. The edited genes are infused into ...