Gene therapy restores immune function in children with rare immunodeficiency
An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to new research. The researchers found that 48 of 50 children who received the gene therapy retained their replenished immune system function two to three years later and did not require additional treatments for their condition, known as ADA-SCID.
Molecular analysis identifies key differences in lungs of cystic fibrosis patients
A team of researchers has developed a molecular catalog of cells in healthy lungs and the lungs of people with cystic fibrosis.
Stem cells create early human embryo structure in advance for fertility research
Stem cells have the ability to turn into different types of cell. Now scientists have developed a method to organize lab-grown stem cells into an accurate model of the first stage of human embryo development.
Restricting growth, spread of head and neck cancers
Researchers have discovered a key molecule that allows cancer stem cells to bypass the body's natural immune defenses, spurring the growth and spread of head and neck squamous cell cancers. Their study, conducted in mice, also demonstrates that inhibiting this molecule derails cancer progression and helps eliminate these stem cells.
Scientists reveal how brain cells in Alzheimer's go awry, lose their identity
Despite the prevalence of Alzheimer's, there are still no treatments, in part because it has been challenging to study how the disease develops. Now, scientists have uncovered new insights into what goes awry during Alzheimer's by growing neurons that resemble -- more accurately than ever before -- brain cells in older patients. And like patients themselves, the afflicted neurons appear to lose their cellular identity.