Gene-editing kicked off 2024 with a bang in the medical sector but closed the year with a whimper. It started with the FDA ...

Sickle cell disease (SCD) is a group of inherited blood disorders resulting from a mutation in the hemoglobin protein in red blood cells. This mutation changes the shape of the blood cells from round, ...

It is a potential game changer for young sickle cell patients because the therapy eliminates the need for a suitable donor and removes the mutation for a life free of sickle cell disease." ...

Study: Different RAS mutations each have unique impact on ... Study: Bone mass declines with age for patients with sickle cell disease Sickle cell disease, a blood disorder that disproportionately ...

Sickle-cell anaemia is the name of a specific form of sickle-cell disease in which there is homozygosity for the mutation that causes HbS. Sickle-cell anaemia is also referred to as "HbSS", "SS ...

Sickle-cell anaemia is the name of a specific form of sickle-cell disease in which there is homozygosity for the mutation that causes HbS. Sickle-cell anaemia is also referred to as "HbSS", "SS ...

Only a few patients have undergone an intensive gene therapy cure for sickle cell disease since it was approved by the FDA due to it's high cost and limited access. NBC News' Yamiche Alcindor spoke ...

The sister of a"caring and selfless” dad whose life was tragically cut short of Christmas Day after a long battle with sickle ...

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 ...

The European Commission has granted orphan drug status to mitapivat, an oral investigational therapy for sickle cell disease.

The first gene-editing treatments for sickle cell, including one powered by CRISPR ... dramatically improved the outlook for people with mutation-driven cancers, yet too often patients still ...