3 promising CRISPR therapies, including one targeting Duchenne Muscular Dystrophy

duchenne muscular dystrophy
Image: Cell Code

With dozens of efforts in the making, from premature aging to obesity and developmental brain disorders, here are the frontrunners beyond CRISPR-based cancer therapy to watch out for.

In February 2019, a team found that a single injection of the CRISPR machinery in mice with [Duchenne Muscular Dystrophy] boosted their muscle function for well over a year.


Hot on DMD’s heels is a CRISPR-based therapy that hopes to eliminate—deep breath—Leber’s congenital amaurosis type 10. Known as LCA10, it’s the most common form of inherited blindness in children. [December 2018], Editas Medicine, along with its partner Allergan, received the FDA green light to start pushing for a phase 1/2 trial

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Scientists are using CRISPR to give [sickle cell anemia] patients’ blood stem cells a protein called HbF, which is a natural protein present at birth that is especially potent at carrying oxygen, which “sickled” red blood cells struggle with.


Of these three non-cancer CRISPR therapies, the first to hit the market will mark a historical turning point for gene therapy and for medicine as a whole: to efficiently edit the very base code that makes us who we are.

Read full, original post: The Three Frontrunners in the CRISPR Therapy Race

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