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World's first in-human gene-editing treatment will tackle hemophilia
Hemophilia B is a terrifying disease. The livers of those suffering from the genetic disorder fail to produce a key protein called Factor IX, which is responsible for clotting blood. Without this protein, they're at constant risk of uncontrollable bleeding, including internally. However, a pair of researchers believe that their novel gene therapy could permanently cure the disease. To that end, the team of Michael Holmes and Thomas Wechsler from Richmond, California's Sangamo biopharmaceuticals, have announced that the world's first in-patient gene-editing therapy targeting these faulty genes will commence next week.
