Thursday, March 14, 2024

Sickle Cell Gene Therapy

 FDA approves two gene cell therapies to treat the disorder of sickle cell anemia. Of these two treatments, the gene-editing CRISPR technology is utilized. In this technology the patient's blood cell's DNA is permanently changed. The CRISPR technology will knock out a gene that triggers the defective blood cells, while medication will kill off the flawed cells. The second approved therapy is called Lyfgenia, which utilizes a common virus that delivers genetic modifications to the patient's blood stem cells in the bone marrow. These modifications cause the blood cells to start producing healthy blood cells. Both therapies involve stem cells being removed from a patient's blood for treatment. 

These new found treatments and technologies are a huge advancement in the medical world. Having these treatments available will helps many have the chance at a life without the pain of the disease. However, the concerns lie with the costs of these treatments. It is likely that these will not be affordable and very expensive. This would mean that many will not be able to receive this treatment and likely will still have to live with the pain from the disease. 

1 comment:

  1. The FDA green-lighting two gene cell therapies for sickle cell anemia is a game-changer! These treatments could mean a life without the constant pain of the disease for so many. But, yeah, the worry about sky-high costs is real. If they're not affordable, what's the point, right? Everyone deserves a shot at better health, no matter their bank account.