Vector image of DNA and lungs on a blue background
|
Research Feature
Could next-generation gene editing mean a cure for cystic fibrosis (CF)? There's been a lot of advancement on the disease in the last decade, but there are still those living with the disease who get no relief from current treatments - especially those for whom health disparities already exist. Paul McCray, M.D., professor of pediatrics, and...

The NHLBI Drives Progress Toward Cures

In December 2023, the U.S. Food and Drug Administration approved two new gene therapies that are transformative therapies for sickle cell disease: 

sickle cell

NHLBI Annual Conference Highlights Recent Advances in Sickle Cell Disease Research and Care 

The yearly NHLBI Sickle Cell Disease Research Meeting is a forum for scientists and healthcare providers to learn about ongoing research in the scientific and clinical aspects of the disease. View recordings of the 3-day, 2024 meeting:

DNA
NHLBI News

In December 2023, the FDA approved two gene therapies for some people with sickle cell disease. Everyone’s experience is different, but people treated with gene therapy tend to have less anemia, fewer health problems related to sickle cell, and better health-related quality of life. This important advance was built upon years of NHLBI-funded discoveries about the genes that cause sickle cell disease and affect disease severity and how best to perform gene therapy. 

Call a healthcare provider or 9-1-1 if a woman is having certain health problems during pregnancy or within 3 months after giving birth. The following symptoms need immediate medical care

  • Trouble breathing 
  • Extreme swelling of hands or face 
  • Thoughts of harming herself or the baby 
  • Changes in vision 

Also seek care right away if the baby’s movements stop or slow, or there is vaginal bleeding or fluid leaking.

Taking Action to Reduce Disparities in Maternal Health

In the United States, pregnant Black women who have sickle cell disease have a risk of dying that is 10 times higher than the risk faced by pregnant Black women who don’t have the disease. The NHLBI is leading research to reduce health disparities that raise the risk of death and serious illness for Black women in the United States, including those who live with sickle cell disease.