CRISPR Gene Editing Used to Cure Rare Genetic Disorder
CRISPR Gene Editing Used to Cure Rare Genetic Disorder
In a historic breakthrough for genetic medicine, scientists have successfully used CRISPR gene editing technology to cure a patient suffering from a rare genetic disorder. The disorder, known as Transthyretin Amyloidosis (ATTR), is caused by a mutation in the TTR gene that leads to the buildup of misfolded proteins in tissues and organs, causing progressive nerve damage and organ failure. This marks the first time CRISPR has been used systemically inside the human body to directly correct a disease-causing gene.
The treatment, developed by biotech company Intellia Therapeutics in collaboration with Regeneron, involves a single intravenous infusion of a CRISPR-based therapy. Unlike earlier gene-editing approaches that required removing cells from the body, editing them in the lab, and reinfusing them, this approach edits the gene in vivo—within the body itself. The therapy uses a lipid nanoparticle to deliver CRISPR components directly to liver cells, where the faulty gene is silenced.
In early-stage clinical trials, patients who received the treatment showed an average 87% reduction in the level of the toxic TTR protein just weeks after treatment. Some saw reductions greater than 95%. More importantly, no serious side effects were reported, and the treatment appears to be durable—potentially offering a permanent solution after a single dose.
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a revolutionary gene-editing tool that allows scientists to precisely cut and modify DNA. Since its development in the early 2010s, CRISPR has held immense promise for treating genetic disorders, but until recently, its use has been largely confined to laboratory and experimental settings.
This success with ATTR is being hailed as a milestone not only for CRISPR but for the future of personalized medicine. It demonstrates the feasibility of using gene editing to treat diseases at their root cause, rather than just managing symptoms. While the therapy is still in clinical trials and broader approval will require more data, the results have sparked optimism for applying CRISPR to other inherited conditions such as sickle cell anemia, cystic fibrosis, and Huntington’s disease.
The achievement also raises important ethical and regulatory questions about the future of gene editing in humans. Nevertheless, it underscores the transformative potential of CRISPR as a tool to cure once-incurable diseases through precise, targeted genetic intervention.