Sickle

Blood sample of patient positive tested for sickle cell.Kitsawet Saethao | Istock | Getty ImagesThe approval of two gene therapies to treat sickle cell disease has given hope to patients who suffer from the debilitating disease, which overwhelmingly affects Black people and people of color.Health officials now face a challenge in finding a way to provide equitable access to the costly treatments.The crippling episodes of pain from the genetic blood disorder make life unpredictable for patients like Michael Goodwin.…

More than 50 years ago my parents took a big chance. They fell in love, got married and had three daughters, not knowing that they both carried the genetic trait for sickle cell disease.When I first started my career in emergency medicine 28 years ago, the main treatments for this disease were intravenous fluids, oxygen and a powerful narcotic used to manage the pain of sickle cell. With the Food and Drug Administration’s recent approval of two treatments for sickle cell that use the gene-editing technology CRISPR, people…

For the first time, the FDA has approved two cell-based gene therapies for treating the rare but potentially fatal sickle cell disease. One is also the first approved therapy to utilize the CRISPR/Cas9 gene editing technology, signifying innovative advancement in the fields of gene therapy and regenerative medicine.Sickle cell disease (SCD) is a group of inherited blood disorders that affect hemoglobin, the oxygen-carrying protein in red blood cells. In someone with SCD, the hemoglobin is mutated, causing the red blood…

CRISPR, the gene-editing technology that has revolutionized biological research, is finally available as a medical treatment with regulatory approval. On December 8 the U.S. Food and Drug Administration approved the first CRISPR treatment for sickle cell disease.The treatment, called exa-cel and made by the companies Vertex and CRISPR Therapeutics, edits a gene involved in red blood cell shape and function. It appears to functionally cure the disease for at least one year. The FDA’s decision makes the U.S. the second…

Recent research suggests that stem cell gene therapy, utilizing CRISPR-Cas9 technology, offers a promising curative treatment for sickle cell disease, marking a significant advancement in the field of gene therapy. New findings from a clinical trial, recently published in the New England Journal of Medicine, suggest that stem cell gene therapy could be a potential curative treatment for sickle cell disease (SCD), a painful, inherited blood disorder. This research adds to the growing evidence that supports gene therapy as…

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…

In a landmark decision, the UK’s approved the use of a gene-editing therapy called for patients with sickle cell disease and beta thalassemia — both of which are hereditary disorders related to genetic mutations of the red blood cells. The treatment, , is the first-ever approved therapy that utilizes CRISPR-based gene editing technology to treat eligible patients.The UK approval of the novel therapy is informed by two previous global clinical trials that indicated the treatment's efficacy. 97 percent of patients using…

The United Kingdom’s…

GARDEN TOOLS are rarely prized for their looks. But recently, elegant iterations of classic shed supplies are showing up in unexpected places. You can find hand-forged trowels at home-decor stores, Japanese digging knives sold alongside selvedge jeans, and carbon-steel floral scissors on Marie Kondo’s website. These new shears, shovels and sickles don’t just appeal for their lovely curves. Make just one cut with a tool of this class, and feel its keen blade pass through branches like…

We always hear about how the Apple Watch can be an ally to help prevent and discover diseases. While it’s a long journey for Apple to say its smartwatch can help with X or prevent Y, important universities conduct studies to see how the wearable could help physicians and patients better understand the disease they want to prevent or treat. This time, a team of researchers from Duke University, Northwestern University, and others published a paper (via MyHealthyApple) showing how the Apple Watch could help…