Gene therapies for sickle cell disease come with hope and challenges

Today, it’s clear that our genes not only cause many diseases, but also hold potential cures. But that wasn’t always the case. It wasn’t until 1949 that scientists first found the molecular culprit of a disease — its roots in the genetic code. The disease was the blood disorder known as sickle cell disease, an inherited disorder that causes severe and debilitating pain. Now, nearly 75 years later, researchers are developing gene therapies to cure it.

Sickle cell disease results from a change in a key protein in hemoglobin, which helps transport oxygen in red blood cells….