In the last issue of Blood, Boulos et al analyze the cost-effectiveness of factor IX gene therapy in patients with severe hemophilia B, in a microsimulation Markov model. Hemophilia is an X-linked disease that affects 400000 people worldwide, of whom 15% have hemophilia B caused by deficiency of factor IX (FIX), which affected the offspring of Queen Victoria,
Scientists at Oxford University have identified the gene responsible for doubling the risk of respiratory failure from COVID-19. Sixty percent of people with South Asian ancestry carry the high-risk genetic signal, partly explaining the excess deaths seen in some UK communities, and the impact of COVID-19 in the Indian subcontinent. Previous work has already identified
Treatment with SP-101, an investigational gene therapy that Spirovant Sciences is developing for people with cystic fibrosis (CF) who are unable to benefit from current treatments, effectively increased CFTR gene activity in a ferret model of the disease, the company reported. Katherine Excoffon, PhD, Spirovant’s vice president of research, presented the data at the 2021 North American Cystic Fibrosis Conference (NACFC). Her
A new study in the leading journal Science reviewed COVID-19 breakthrough infections among 780,225 Veterans, finding that vaccine protection declined from 87.9% to 48.1% during the 2021 Delta surge in the U.S. The researchers from PHI, the Veterans Affairs Medical Center and the University of Texas Health Science Center found a dramatic decline in effectiveness
In March 2021, 1,480 U.S. businesses operating 2,754 clinics were found selling purported stem cell treatments for various indications. More than four times as many businesses than were identified 5 years ago are selling stem cell products that are not FDA-approved and lack convincing evidence of safety and efficacy following a new paper published in Cell
Nearly two years since becoming a global pandemic that has killed millions of people, the mystery of which proteins in the SARS-CoV-2 virus are responsible for severe vascular damage that could even lead to heart attack or stroke has not yet been solved. Now, for the first time, a team of experts led by Tel
In a breakthrough for the treatment of aggressive solid cancers, researchers at Children’s Hospital of Philadelphia (CHOP) have developed a novel cancer therapy that targets proteins inside cancer cells that are essential for tumor growth and survival but have been historically impossible to reach. Using the power of large data sets and advanced computational approaches,
Mesenchymal stem cells (MSCs) are multi-functional stem cells that are present in multiple human tissues and can be found in the spinal cord, umbilical cord blood, umbilical cord tissue, placenta tissue, adipose tissue, etc.. With low immunogenicity, multi-directional diferentiation ability, in particular homing ability, MSCs have signifcant research potential in cardiovascular diseases, nervous diseases, and
Treatment with mesenchymal stem cells (MSCs) has failed to significantly reduce inflammation in the brain of adults with multiple sclerosis (MS) in a phase 2 clinical trial. The into-the-vein treatment also failed to improve other clinical aspects of the condition. The results were shared in a study titled “Safety, tolerability, and activity of mesenchymal stem cells versus placebo
Imagine knee replacement surgery with a “smart” implant that collects and transmits data enabling an orthopedic surgeon to monitor a patient’s recovery from afar. It became a reality this week when Peter Sculco, MD, and Fred Cushner, MD, orthopedic surgeons at Hospital for Special Surgery (HSS) performed the first knee replacement containing a smart sensor capable of
