Advisers to the Food and Drug Administration meeting Tuesday paved the way for the first treatment of human disease using the gene-editing technique CRISPR. The agency has a December deadline.
"It's really life-changing," says Victoria Gray, when describing the gene-editing treatment for sickle cell disease that she received as part of a clinical trial in 2019.The Food and Drug Administration appears poised to approve the first medical therapy that uses gene-editing to treat a disease.
The treatment worked in 29 of 30 patients followed for at least 18 months, and doesn't appear to cause any serious short-term safety concerns, the company reported. "I believe if you say. 'Yes,' to this treatment that it's going to change the lives positively of many people who are suffering from diseases and disorders who now feel hopeless," Gray said."Once it comes, they can feel hope again, just like I did."
For the treatment, doctors remove cells from each patient's bone marrow, edit a gene with CRISPR and then infuse billions of the modified cells back into patients. The edited cells produce a form of hemoglobin known as fetal hemoglobin, restoring normal red blood cell function. While not a cure for the disease, the hope is exa-cel will be a one-time treatment that will alleviate symptoms for a lifetime.
Many of the countries where most sickle cells patients live don't have enough sophisticated medical centers to provide the complicated treatment, Creary notes.
South Africa Latest News, South Africa Headlines
Similar News:You can also read news stories similar to this one that we have collected from other news sources.
Some Cold Medicines Don’t Work. Here’s What Doctors Recommend Instead.With FDA advisers determining that phenylephrine is ineffective, doctors and pharmacists recommend alternatives for congestion relief
Read more »
More than 1,000 pay tribute to Maine's mass shooting victims on day of prayer, reflection on tragedyYork Opioid Collaborative joined forces with the Drug Enforcement Administration for National Drug Take Back Day.
Read more »
Hurricane Otis death toll rises to 43, number of missing now 36 as search and recovery work continuesYork Opioid Collaborative joined forces with the Drug Enforcement Administration for National Drug Take Back Day.
Read more »
Recall alert: FDA issues warning over elevated lead levels in WanaBana apple puree pouchesThe Food and Drug Administration has issued a warning concerning WanaBana Apple Cinnamon Fruit Puree Pouches.
Read more »
Recall alert: FDA issues warning over elevated lead levels in WanaBana apple puree pouchesThe Food and Drug Administration has issued a warning concerning WanaBana Apple Cinnamon Fruit Puree Pouches.
Read more »
Fired fast-food worker still has a taste for the food: Bainbridge Township police blotterA Solon man made a scene in the parking lot of his former employer Oct. 17. A patron of the fast-food restaurant told employees inside that the agitated man was waving what appeared to be a gun. He left and police were informed of the incident the following day. The subject had been fired from his job there.
Read more »