Lifeboat Foundation

As a therapy for vision impairment resulting from inherited retinal degeneration, the mRNA would instruct cells in the retina, which are impaired because of a genetic mutation, to manufacture the proteins needed for sight. Inherited retinal degeneration, commonly abbreviated to IRD, encompasses a group of disorders of varying severity and prevalence that affect one out of every few thousand people worldwide.

An example of a genetic pulmonary condition is cystic fibrosis, a progressive disorder that results in persistent lung infection and affects 30,000 people in the U.S., with about 1,000 new cases identified every year. One faulty gene—the cystic fibrosis transmembrane conductance regulator, or CFTR—causes the disease, which is characterized by lung dehydration and mucus buildup that blocks the airway.

The thiophene-based LNP study, which involved mice and non-human primates, stems from a $3.2 million grant from the National Eye Institute. The grant’s purpose is addressing limitations associated with the current primary means of delivery for gene editing: adeno-associated virus, or AAV.

Lymphedema is a chronic condition that affects many cancer survivors. It’s swelling caused by a buildup of lymph fluid, often as a result of cancer treatment. While it can’t be prevented, treatment can help to relieve swelling and improve the ability to function day to day.

Lymphedema is a side effect of some cancer treatments. Learn about symptoms and ways you can manage and treat swelling in your arm or leg caused by

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A significant reduction of GABA and monoamine oxidase B (MOAB), the latter of which is an astrocytic enzyme that produces GABA, was also observed in Crym KO mice. These observations suggest that increased synaptic excitation from IOFC terminals leads to lower levels of tonic GABA, which causes reduced presynaptic inhibition.

Study significance

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Durable remission was achieved with front-line dasatinib plus blinatumomab in adults with Philadelphia chromosome-positive acute lymphoblastic leukemia.

NEJM Journal Watch reviews over 250 scientific and medical journals to present important clinical research findings and insightful commentary.

For decades, scientists and pathologists have tried, without much success, to come up with a way to determine which individual lung cancer patients are at greatest risk of having their illness spread, or metastasize, to other parts of the body.

Now a team of scientists from Caltech and the Washington University School of Medicine in St. Louis has fed that problem to artificial intelligence (AI) algorithms, asking computers to predict which cancer cases are likely to metastasize. In a novel pilot study of non-small cell lung cancer (NSCLC) patients, AI outperformed expert pathologists in making such predictions.

These predictions about the progression of lung cancer have important implications in terms of an individual patient’s life. Physicians treating early-stage NSCLC patients face the extremely difficult decision of whether to intervene with expensive, toxic treatments, such as chemotherapy or radiation, after a patient undergoes lung surgery. In some ways, this is the more cautious path because more than half of stage I–III NSCLC patients eventually experience metastasis to the brain. But that means many others do not. For those patients, such difficult treatments are wholly unnecessary.

Colchicine reduced cardiovascular events in patients with diabetes and recent myocardial infarction in a randomized study.

NEJM Journal Watch reviews over 250 scientific and medical journals to present important clinical research findings and insightful commentary.

A Tufts School of Medicine researcher is developing a test to predict lung cancer risk more accurately and reduce the number of scans to detect the condition.

Cancer cells are characterized by their aggressiveness: they grow rapidly and spread to other parts of the body. To enable this, numerous mechanisms come into play, and one of them involves a protein called MYC, which activates certain genes on the cancer cell’s DNA strand, causing the cancer cell to grow and divide.

The MYC protein is also present in healthy individuals, where it plays a crucial role in regulating many cell functions.

“When cancer occurs, it is due to an accumulation of mutations in our DNA, often resulting in the overactivation of the MYC protein. Therefore, this protein plays a crucial role in most cancer forms,” says Rasmus Siersbæk, head of research at the Department of Biochemistry and Molecular Biology, University of Southern Denmark.

With a mobile app powered by artificial intelligence (AI), Caitlin Hicks, MD, MS, reviews selfies of patients’ feet in real time to track their wounds as part of a clinical trial. The app saves time for Hicks, a vascular surgeon at Johns Hopkins Medicine, but also reduces clinic trips for her patients with diabetes in inner-city Baltimore, many of whom are elderly and less mobile or have other socioeconomic barriers to care. Hicks knows that for these patients, wound vigilance is the linchpin to preventing infection, hospitalization, or, worse, amputation or even death.

Despite their crushing toll, diabetic foot infections remain stubbornly hard to treat, but multidisciplinary care teams, new drugs and devices on the horizon, and practical solutions to socioeconomic factors could budge the needle.