The FDA has approved the world’s first gene-edited treatment for symptoms of sickle cell disease. Experts weigh in on the potential benefits and limitations.

In sickle cell disease, a gene mutation causes the hemoglobin molecules in red blood cells to form a rigid chain. This causes the cells to stiffen into a sickle shape, clogging blood vessels and ...

Sickle cell disease is a group of inherited disorders that affect the hemoglobin, which causes sickle-shaped red blood cells. Learn about the symptoms, causes and treatment.

To safely target BCL11A for sickle cell disease, the team members identified a region within the gene that’s crucial for BCL11A expression specifically in red blood cells.

A person inherits sickle cell disease when they receive two sickle cell genes, one from each parent. Learn who can inherit this condition and how it passes from parents to children.

The switch from fetal γ-globin to adult β-globin is controlled by BCL11A. Genetic manipulation of BCL11A improves disease outcomes by turning γ-globin expression back on to compensate for ...

In sickle cell disease, red blood cells become "sickle" or crescent-shaped due to a genetic mutation affecting hemoglobin -- a protein that carries oxygen from the lungs to other tissues.

Given the extreme neglect of sickle cell disease to date, the sheer number of people affected and all we could learn by studying the illness, we urgently need a global initiative to advance ...

The decision has ushered in a new era for sickle cell disease treatment — and marked the world’s first approval of a medicine based on CRISPR/Cas9 gene-editing technology.

UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...

Sickle cell disease is an autosomal recessive disorder caused by mutations in the hemoglobin β gene (HBB), which encodes adult hemoglobin. The most common mutation replaces glutamic acid with valine ...