therapies

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.…

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…

A groundbreaking study at UMass Amherst has decoded how sugars attached to proteins guide their correct folding, shedding light on potential treatments for diseases caused by protein misfolding. This protein (red) has been glycosolated with glycans (blue and green). Credit: UMass Amherst Team’s approach reveals crucial role played by a specific enzyme in the folding process. While we often think of diseases as caused by foreign bodies—bacteria or viruses—there are hundreds of diseases affecting humans that result from…

Around a million Americans are living with Parkinson’s disease, the neurological condition that greatly, and most often progressively, affects the dopamine-producing powerhouse area of the brain, the substantia nigra. While there is no cure, medication is generally aimed at substituting or boosting dopamine, which can help lessen the movement-impairment that comes with the debilitating disease.Studies recently have pivoted towards investigating specific cellular mechanisms involved in the disease’s progression, such as…

From penicillin to rapamycin, the route to discovering remarkable medicines has often been a fortuitous one. Now, researchers are hoping that another surprise find, once again linked to bacteria, can be harnessed for its medical potential and even be used to destroy cancer cells.In a new study, scientists from the University of Queensland have found that the venom found in the bristles of the asp caterpillar (Megalopyge opercularis) can punch holes in cells in the same way that the sickness-causing E. coli and Salmonella…

A new research study led focused on a specific E. coli subset known as K1, which has the potential to cause serious diseases including bloodstream infections, kidney infections, and meningitis in newborns. They found that the virulent K1 capsule’s existence predates previous estimations by about 500 years, emphasizing its role in E. coli‘s survival and pathogenicity.Researchers have discovered that a specific virulent E. coli subset, K1, predates previous estimations by around 500 years and 25% of current E. coli strains…

A researcher in the lab of Rice’s Angel Martí holds a vial of fluorescent dye molecules in solution. Using time-resolved spectroscopy, which tracks the fluorescence lifetime of dye molecules, Martí and collaborators describe a second binding site on amyloid-beta deposits associated with Alzheimer’s disease, opening the door to the development of new therapies. Credit: Gustavo Raskosky/Rice UniversityThe research has uncovered that the protein plaques associated with Alzheimer’s are stickier than previously…

Before 2020, you’d be hard-pressed to have found talk of mRNA therapies in the media. And while not a new technology, having been identified in 1961, it was thrown into the spotlight with the lightning-fast rollout of COVID vaccines, heralded as an incredible scientific feat and simultaneously maligned as haphazard experimental medicine.The vaccines were the first broadly successful therapy using mRNA technology, but researchers have been trying to master targeted treatments for decades, too. Now scientists believe…

Meredith Broussard is unusually well placed to dissect the ongoing hype around AI. She’s a data scientist and associate professor at New York University, and she’s been one of the leading researchers in the field of algorithmic bias for years. And though her own work leaves her buried in math problems, she’s spent the last few years thinking about problems that mathematics can’t solve. Broussard argues that we are consistently too eager to apply artificial intelligence to social problems in inappropriate and damaging…

I watched scientists, ethicists, patient advocacy groups, and others wrestle with these topics at the Third International Summit on Human Genome Editing in London earlier this week. There’s plenty to get excited about when it comes to gene editing. In the decade since scientists found they could use CRISPR to edit cell genomes, multiple clinical trials have sprung up to test the technology’s use for serious diseases. CRISPR has already been used to save some lives and transform others. But it hasn’t all been smooth…