Diseases, Conditions, Syndromes
CRISPR gene editing reveals biological mechanism behind common blood disorder
Asymptomatic sickle cell disease patients actually lack a tiny part of the genome, UNSW scientists have shown.
Apr 8, 2022
0
66
Page 5 - News tagged with sickle cell gene
Diseases, Conditions, Syndromes
A possible cure for sickle cell? High-tech solution unlikely to help patients with limited access
Sickle cell anemia is an inherited blood disorder where red blood cells become sickle/crescent shaped. It causes frequent infections, swelling in the hands and legs, pain, severe tiredness and delayed growth or puberty. Treatment ...
Feb 16, 2022
0
4
Oncology & Cancer
What caused the blood malignancies linked to gene therapy for sickle cell disease?
Gene therapy trials for sickle cell disease have been showing great promise, even offering hope of a cure. But in early 2021, the trials ground to a halt after reports of blood malignancies in two people in a trial sponsored ...
Feb 15, 2022
0
12
Diseases, Conditions, Syndromes
Could gene therapy help cure sickle cell disease?
A gene therapy that could provide a permanent cure for sickle cell disease continues to show success through a third wave of patients, researchers report.
Dec 13, 2021
0
6
Diseases, Conditions, Syndromes
Understanding sickle cell disease
Sickle cell disease, which is a group of inherited red blood cell disorders, affects approximately 100,000 people in the U.S., according to the Centers for Disease Control and Prevention.
Sep 10, 2021
0
6
Medical economics
New gene therapies may soon treat dozens of rare diseases, but expense may limit availability for many
Zolgensma—which treats spinal muscular atrophy, a rare genetic disease that damages nerve cells, leading to muscle decay—is currently the most expensive drug in the world. A one-time treatment of the life-saving drug ...
Sep 1, 2021
0
16
Diseases, Conditions, Syndromes
Sickle cell plagues many Black Americans, but there's hope for better treatments
It's been more than six months since Brandy Compton last landed in a hospital emergency room.
Jun 21, 2021
0
2
Medical research
Using base editing to treat sickle cell disease in mice
A team of researchers from the Broad Institute, Harvard University and St. Jude Children's Research Hospital has used a base editing technique in a novel way to treat sickle cell disease (SCD) in mice. In their paper published ...
Medical research
Researchers develop proof-of-concept treatment that elevates adult and fetal hemoglobin
Researchers at Children's Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production ...
Jun 1, 2021
0
6
Medical research
Tweaking gene therapy: Scientists experimentally boost red blood cells to aid sickle cell and other hemoglobin diseases
A series of laboratory studies is underway in the United States to improve gene therapy worldwide for sickle cell disease, a complex and sometimes deadly heritable blood disorder that dramatically affects the structure and ...
