CRISPR-Cas9, whose creators were awarded the 2020 Nobel Prize in chemistry a mere eight years after its discovery, allows researchers to alter the DNA of living things at will.
Low-angle view of KSQ Therapeutics, Lab | Central, CASEBIA Therapeutics, Bayer | Life Hub Boston, and CRISPR Therapeutics logos on an Osborn Triangle sign in Cambridge, Massachusetts, February 13, 2021. Photographer credit Nicole Gaffney. Paddy Doherty remembers his father as a proud, hard-working family man who stayed physically fit for most of his life.
"Patients left untreated with this type of amyloidosis develop heart failure, low blood pressure, horrible bowel disturbance, and eventually become incontinent of urine and feces," said Julian Gillmore, nephrologist and head of the National Amyloidosis Centre at University College London. "It's a truly awful, gradually progressive disease that is ultimately fatal."
Now, a growing number of researchers like Gillmore are taking on the challenge to develop CRISPR-based therapies to improve the lives of patients with rare conditions such as cystic fibrosis, Duchenne muscular dystrophy and Huntington's disease that are caused by mutations in single genes. Almost 1 in 10 people in the United States lives with a rare condition of some kind, most of which have a genetic basis with no treatment approved by the Food and Drug Administration available.
For both diseases, CTX001 involves removing blood stem cells from patients' bone marrow, turning on a gene that produces usable hemoglobin and infusing the cells back in. After the therapy, the researchers saw marked improvements in both disease groups for anywhere from three to 26 months of follow-up. All 15 patients with beta-thalassemia, who were previously dependent on blood transfusions, no longer needed them.
Because circulating transthyretin is made almost entirely in the liver - and everything that enters the bloodstream is carried to the liver to metabolize - the researchers realized they could simply inject patients with the CRISPR-based therapy. Patients with other kinds of rare diseases would need a CRISPR-based drug that distributes itself throughout the entire body.
Recent research that created a tailor-made drug within less than a year for a 6-year-old girl with a rare, fatal neurodegenerative disorder inspired him to attempt the same feat for Duchenne. He rallied a group of gene therapy scientists to work on a CRISPR-based therapy for Terry's specific mutation.
His laboratory is testing a CRISPR-based platform that transforms red blood cells into factories for proteins missing in the blood. Preliminary results in mice appear encouraging for rare conditions like hemophilia B, Hurler syndrome, Fabry disease and Wolman disease.
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