Sickle cell cure news

There have been several recent developments and breakthroughs in the search for a cure for sickle cell disease. Here are some of the latest news and updates:

1. Gene editing therapy: In 2020, the FDA approved the first gene editing therapy for sickle cell disease, called Zynteglo (betibeglogene autotemcel). This therapy uses CRISPR-Cas9 gene editing to modify the HBB gene, which causes sickle cell disease. The therapy has shown promising results in clinical trials, with patients experiencing significant reductions in sickle cell crises and improvements in quality of life.
2. Stem cell transplantation: Researchers have made progress in using stem cell transplantation to cure sickle cell disease. A study published in the New England Journal of Medicine found that stem cell transplantation from a matched donor can cure sickle cell disease in up to 90% of patients.
3. Gene therapy: Another gene therapy, called LentiGlobin (LentiGlobin BB305), has shown promising results in clinical trials. This therapy uses a lentivirus to deliver a healthy copy of the HBB gene to the patient's bone marrow, which can help to correct the genetic mutation that causes sickle cell disease.
4. CRISPR-Cas9 therapy: Researchers have also been exploring the use of CRISPR-Cas9 gene editing to correct the genetic mutation that causes sickle cell disease. A study published in the journal Nature Medicine found that CRISPR-Cas9 therapy can effectively correct the mutation in human cells.
5. New treatment approaches: Researchers are also exploring new treatment approaches, such as using small molecules to correct the genetic mutation that causes sickle cell disease. A study published in the journal Science Translational Medicine found that a small molecule called CRISPR-Cas9 can effectively correct the mutation in human cells.
6. Cure rates: According to the Sickle Cell Disease Association of America, cure rates for sickle cell disease have increased significantly in recent years. The organization reports that cure rates have increased from 10% to 20% in the 1990s to 50% to 60% today.
7. Increased funding: There has been an increase in funding for sickle cell disease research in recent years. The National Institutes of Health (NIH) has increased funding for sickle cell disease research, and private organizations such as the Sickle Cell Disease Association of America have also increased their funding for research and treatment.

Overall, while there is still no cure for sickle cell disease, there have been significant advances in recent years, and researchers are hopeful that a cure may be possible in the near future.