Without treatment, he would die. After a desperate search, his family found a promising but risky option: an experimental drug that would edit his DNA inside his body to stop his disease, which is called transthyretin amyloidosis.
[73-year-old Akintunde] Odunsi decided it offered his best chance. “Go for it,” he told himself.It sounds like science fiction, but Odunsi is among dozens of people participating in studies on a controversial new forefront of the gene-editing revolution.
Regulators last year approved the world’s first medicine using Crispr, the Nobel Prize-winning tool for modifying genes. The medicine, for sickle-cell disease, a group of inherited blood disorders, involves extracting cells, editing them in a lab and putting them back in the patient’s body.
“In vivo” gene editing, as the approach is called, could transform medicine. Several of the therapies are for cardiovascular disease, and if proven safe and effective could reach millions of patients.
Related on the SLP
…
In vivo editing could be less expensive and reach more people than editing cells outside the body. It doesn’t require the laboratories and expertise needed to extract and edit cells.
Editing inside the body might also be easier on patients. They don’t have to undergo chemotherapy, for example, which is necessary for sickle-cell patients before receiving their cells that have been edited outside the body.
