Could a CRISPR Therapeutic Cure SCD

Christopher Vakulskas, PhD believes CRISPR Therapy can. At present, his group is focused on new CRISPR technologies with emphasis on Cas9 from Streptococcus pyogenes and Cas12a (Cpf1) from Acidaminococcus species. He also leads work to evaluate and reduce CRISPR off-target cleavage events.

For many serious diseases, the statistics tell just half the story. Sickle-cell disease (SCD) is no exception. Yes, we can say that SCD affects approximately 100,000 people in the United States and thus qualifies as the nation’s most common inherited blood disorder.1 And we can note that around the world, about 300,000 babies are born each year with the disease.2 But even numbers as grim as these fail to convey what SCD does to people at the individual level.

To get a sense of the humanity behind the statistics, visit “Real Stories from People Living with Sickle Cell Disease,” a webpage posted by the Centers for Disease Control and Prevention.3 Read about patients with SCD who experience anemia, chronic pain, repeated infections, and other health complications. Read about families that care for loved ones struggling with SCD. Read about healthcare professionals who are devoted not only to their patients, but also to raising awareness about SCD management.

https://www.genengnews.com/commentary/point-of-view/could-a-crispr-therapeutic-cure-sickle-cell-disease/