geneediting

Many CRISPR treatments are in trials, but in 2022, Vertex Pharmaceuticals, based in Boston, was first to bring one to regulators for approval. That treatment was for sickle-cell. After their bone marrow was edited, nearly all the patients who volunteered in the trial were pain free.  Good news. But the expected price tag of the gene-editing treatment is $2 to $3 million. And Vertex has no immediate plans to offer it in Africa—where sickle-cell disease is most common, and where it still kills children. The company says…

It originates in the bone marrow due to a genetic kink that inhibits the production of healthy hemoglobin and is associated with devastating, lifelong, and potentially fatal health consequences, including stroke, increased risk of infections, painful episodes classified as “vaso-occlusive crises” that require emergency medical attention, liver problems, heart problems, and eye problems, according to the National Institutes of Health (NIH). There are an estimated 100,000 people in the U.S., 15,000 in the U.K., and 20…

Blood sample tube with blood for Sickle Cell Screen -Test. Sickle cell blood test, abnormal hemoglobin test.Md Babul Hosen | Istock | Getty ImagesThe U.S. Food and Drug Administration on Friday approved the country's first gene-editing treatment, Casgevy, for use in patients with sickle cell disease.The approval comes about a decade after the discovery of CRISPR technology for editing human DNA, representing a significant scientific advancement. Yet reaching the tens of thousands of people who could benefit from the…

—By Amba Kak, Sarah Myers West and Meredith Whittaker, members of the AI Now Institute Until late November, when the epic saga of OpenAI’s board breakdown unfolded, the casual observer could be forgiven for assuming that the ecosystem around generative AI was vibrant and competitive.But this is not the case—nor has it ever been. And understanding why is fundamental to understanding what AI is, and what threats it poses. Put simply, in the context of the current paradigm of building larger- and larger-scale AI systems,…

I’m very aware of how privileged I am to have been an early recipient and to reap the benefits of this groundbreaking new treatment. People with sickle cell disease don’t produce healthy hemoglobin, a protein that red blood cells use to transport oxygen in the body. As a result, they develop misshapen red blood cells that can block blood vessels, causing intense bouts of pain and sometimes organ failure. They often die decades younger than those without the disease. After I received exa-cel, I started to experience…

CRISPR systems are powerful tools for genetic engineering, but they have their limitations. Now, scientists have discovered almost 200 new CRISPR systems in their native habitat of bacteria, and found that some can edit human cells even more precisely than existing ones.The CRISPR-Cas9 gene-editing tool is one of the most important scientific developments of the past decade, earning its discoverers a Nobel Prize in Chemistry. Scientists can use it to make efficient cut-and-paste edits to human cells, potentially treating…

Last week, in a global milestone for medicine, U.K. regulators approved the first-ever CRISPR gene-editing drug. The treatment, from Vertex and CRISPR Therapeutics, will be licensed under the brand name Casgevy in the U.K. for patients with sickle cell disease (SCD) and beta thalassemia. The regulatory green light abroad isn’t surprising given the therapy’s established record of clinical trial success in treating these painful, and sometimes fatal, inherited blood disorders. But it marks a historic turning point for…

The UK has become the first country in the world to approve CRISPR gene-editing therapy. The landmark biotech decision involves the treatment of two specific blood dieseases, but also opens the door for the use of the technology in treating many other genetic disorders.  Regulators approved the use of CRISPR for the treatment of inherited diseases sickle-cell anaemia and β-thalassaemia on Thursday. The former affects the shape of red blood cells of 20 million people worldwide and can cause…

In a trial run by , a Cambridge–based biotech company, researchers discovered that a single infusion of a gene-editing treatment called VERVE-101 was able to reduce cholesterol levels in patients. This treatment was tested in individuals with hereditary conditions that made them susceptible to developing clogged arteries and heart attacks. Scientists were able to use techniques to tweak liver gene cells. The researchers a cholesterol-raising gene called PCSK9, which is found in the liver, in order to lower — sometimes…

Researchers have presented the interim results of a clinical trial that used a single infusion of CRISPR gene-editing technology to permanently switch off low-density lipoprotein cholesterol production in people with a genetic condition that causes elevated levels of the ‘bad’ cholesterol and increases the risk of early heart attack.Low-density lipoprotein cholesterol (LDL-C), so-called ‘bad’ cholesterol, is known to be a primary driver of atherosclerosis, the build-up of plaque on the walls of blood vessels that places…