CRISPR is a technique of gene editing which has already won its inventors the Nobel Prize.
Great progress has been made. CRISPR is now being used to cure genetic diseases.
By Fyodor Urnov, The New York Times, Dec. 9, 2022
Gene editing is much newer technology and builds on the gains of gene therapy. Instead of using a virus, however, gene editing relies on a molecular machine called CRISPR, which can be instructed to repair a mutation in a gene in nearly any life form, right where that “typo” occurs. Impressively versatile, potential applications for CRISPR range from basic science to agriculture and climate change. In medicine, CRISPR gene editing allows physicians to directly fix typos in the patients’ DNA. And so much substantive progress has been made in the field of genetic medicine that it’s clear scientists have now delivered on a remarkable dream: word-processor-like control over DNA.
The first person to be gene-edited with CRISPR was treated only three years ago for a disorder of red blood cell production, and since then, the technology has been used to treat congenital blindness, sickle cell disease, heart disease, nerve disease, cancer and H.I.V. While not all diseases have a single-gene basis, most have a genetic component. Early studies suggest that conditions like heart disease, chronic pain and Alzheimer’s disease could all be treated with CRISPR…
The biotechnology companies CRISPR Therapeutics and Vertex have cured 31 people with sickle cell disease, who no longer experience the debilitating episodes of pain that characterize their condition. Another biotechnology company, Intellia Therapeutics, teamed up with Regeneron and used CRISPR to inactivate a typo-laden toxic gene in the livers of 15 people. A mere month after this injection, 93 percent of the toxin was gone from the bloodstreams of patients who received the highest dose of CRISPR medicine. Verve Therapeutics is developing a CRISPR treatment for heart disease, with an initial focus on a severe genetic form…
There are up to 400 million people worldwide affected by one of the 7,000 diseases caused by mutations in single genes. Scientists owe them and their families honesty about the chasm between a test tube in a lab and an IV line in a hospital. The greatest obstacles are not technical but legal, financial and organizational… [end quote]
CRISPR is in clinical trial for correcting muscular dystrophy. CRISPR is also being studied in cystic fibrosis. These deadly diseases have killed people I knew personally.
Gene therapy is very, very expensive since it is still in the experimental phase. But it’s very early days since CRISPR was only invented about 10 years ago. With funding provided by the organizations dedicated to common, specific genetic diseases (remember the Jerry Lewis Telethon for muscular dystrophy?) those will probably reach FDA approval and widespread clinical use earliest.