Researchers reported promising outcomes data for the first group of boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a fatal genetic immunodeficiency also known as "bubble boy" disease, who were treated as part of an international clinical study of a new form of gene therapy.
Researchers have identified a protein that causes loss of function in immune cells combatting HIV. The scientists report that the protein, Sprouty-2, is a promising target for future HIV drug development.
A novel immune-boosting drug combination eradicates glioblastoma brain cancer in mice, according to a new study.
Chemical engineers have developed a novel way to generate nanoparticles that can recognize specific molecules, opening up a new approach to building durable sensors for many different compounds.
A single overactive enzyme worsens the two core defects of diabetes—impaired insulin sensitivity and overproduction of glucose—suggesting that a drug targeting the enzyme could help correct both at once, according to new mouse studies.
A new nanotechnology-based technique for regulating blood sugar in diabetics may give patients the ability to release insulin painlessly using a small ultrasound device, allowing them to go days between injections.
After more than four years of investigation, researchers have discovered a promising treatment for osteoporosis, which is easily delivered in water soluble form.
The deadliest brain cancer requires grueling treatment with bleak prospects for survival. Now, researchers have discovered a key component to how these aggressive tumors grow that could lead to better solutions.
Princeton University officials decided Monday to make available a meningitis vaccine that hasn't been approved in the U.S. to stop the spread of the sometimes deadly disease on campus.
Researchers have developed a new technique for fighting deadly and hard-to-treat pancreatic cancer that uses two different types of nanoparticles, the first type clearing a path into tumor cells for the second, which delivers chemotherapy drugs.
The transplant anti-rejection drug rapamycin showed unexpected benefits in a mouse model of a fatal defect in the energy powerhouses of cells, the mitochondria.
Friendly microbes in the intestinal tracts (guts) of healthy American children have numerous antibiotic resistance genes, according to results of a pilot study by scientists.
Researchers studying how cancer spreads into bone have made a surprising discovery that suggests several investigational anti-cancer therapies just entering the drug-development pipeline may not have the desired effect.
U.S. experts are raising the alarm over the spread of drug-resistant malaria in several Southeast Asian countries, endangering major global gains in fighting the mosquito-borne disease that kills more than 600,000 people annually.
A team of scientists have identified a novel therapeutic approach for the most frequent genetic cause of ALS, a disorder of the regions of the brain and spinal cord that control voluntary muscle movement, and frontotemporal degeneration, the second most frequent dementia.
Researchers have launched an early-stage clinical trial of an investigational vaccine designed to prevent genital herpes disease.
The generic anticonvulsant medication gabapentin shows promise as an effective treatment for alcohol dependence, based on the results of a 150-patient clinical trial of the medication. The study found that alcohol dependent patients using gabapentin were more likely to stop drinking or refrain from heavy drinking than those taking placebo.
An experimental vaccine to protect against respiratory syncytial virus (RSV), a leading cause of illness and hospitalization among very young children, elicited high levels of RSV-specific antibodies when tested in animals, according to a new report.
Cisplatin is a chemotherapy drug given to more than half of all cancer patients. The drug kills cells very effectively, but tumors can become resistant to the drug and grow back. A new study offers a possible way to overcome that resistance.
A bone drug already on the market for osteoporosis may kill chronic myelogenous leukemia (CML) stem cells thought to persist in the bone marrow after standard therapy, lowering the likelihood of disease recurrence, according to a new study.
A team of scientists created and characterized a new molecule, using elements of Clostridium botulinum and Clostridium tetani neurotoxins, that was able to alleviate hypersensitivity to inflammatory pain.
A new type of “good cholesterol,” made in the lab, could one day deliver drugs to where they are needed in the body to treat disease or be used in medical imaging, according to scientists.
Two subtypes of human papillomavirus (HPV) prevented by vaccines are half as likely to be found in African-American women as in white women with precancerous cervical lesions, according to researchers.
In a pair of landmark studies that exploit the genetic sequencing of the “missing link” cold virus, rhinovirus C, scientists have constructed a 3-D model of the pathogen that shows why there is no cure yet for the common cold.
Mice models of Alzheimer's treated with one abdominal injection of an antibody fragment designed by researchers showed, after only five days, improved memory and ability to learn as the result of less aggregated toxins and an increase in the number of neurons.