Gene therapy having big sickle-cell disease results


Image result for These Patients Had Sickle-Cell Disease. Experimental Therapies Might Have Cured Them.

Success against sickle-cell would be “the first genetic cure of a common genetic disease” and could free tens of thousands of Americans from agonizing pain.

Researchers are trying to address sickle-cell disease at the genetic level, and it’s having drastic results so far among the patients participating in clinical trials, the New York Times reports.

  • The experimental gene therapy treatments are still in their early stages, and it could be at least 3 years before one is approved. But a handful of the enrollees no longer show signs of the disease.
  • Currently, the only way to treat those with sickle-cell is through a bone marrow transplant, which is dangerous, expensive and uncommon.

The bottom line: This would be the first genetic cure of a common genetic disease,” Dr. Edward Benz, a professor at Harvard Medical School, told the NYT.

Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out /  Change )

Google photo

You are commenting using your Google account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s

This site uses Akismet to reduce spam. Learn how your comment data is processed.