Body: Duchenne muscular dystrophy (DMD) is a rare but devastating genetic disorder that causes muscle loss and physical impairments. Researchers have shown in a mouse study that the powerful gene editing technique known as CRISPR may provide the means for lifelong correction of the genetic mutation responsible for the disorder.
Body: A study carried out with a new human stem cell-derived model reveals that the most prevalent genetic risk factor of Alzheimer's disease (AD), apolipoprotein E4 (APOE4), impairs the function of human brain immune cells, microglia. These findings pave the way for new, effective treatment approaches for AD.
Body: Scientists have unraveled parts of a mechanism that may one day help to treat Rett syndrome and other genetic disorders linked to the X chromosome.
Body: Doctors have successfully treated an infant with a rare childhood leukemia using a targeted therapy approved for adults with inoperable liver cancer and advanced kidney cancer. The decision to use the drug, sorafenib, was made after pathologists identified a unique mutation of two genes being fused together instead of on separate chromosomes.
Body: Study finds that the diabetes drug metformin, known to promote brain repair, can also help restore cognitive function in adult mice but only in females and in a way that is dependent on the sex hormone estradiol.
Body: Twenty people die waiting for an organ transplant every day in the US, but lab-grown organs so far lack the cellular density, vasculature, and functions required to make them viable replacements. The new SWIFT method solves those problems by 3D printing vascular channel networks directly into living organ building blocks, enabling the creation of larger tissues approximating the size and function of organs, including heart tissue that beats on its own over a seven-day period.
Body: The neurobiological pathophysiology of schizophrenia differs significantly between males and females, according to a new study. The findings suggest a possible need for more sex-specific treatments for schizophrenia. The study was the first to identify a number of sex-specific genes related to schizophrenia using neurons derived from induced pluripotent stem cells.
Body: New research reveals a new role for the enzyme telomerase, which scientists thought was turned off in most normal adult cells, except in cancerous tumors where it promotes unlimited cell division. The researchers discovered that as normal, healthy adult cells approach cell-death, they produce a burst of telomerase that prevents malignancies and softens the final steps in the aging process.
Body: Scientists have created miniature brains from stem cells that developed functional neural networks. Despite being a million times smaller than human brains, these lab-grown brains are the first observed to produce brain waves that resemble those of preterm babies.
Body: The expression of many genes that have previously been associated with autism is abnormal also in violent psychopathy, a new study shows. The researchers used stem cell technology to analyse the expression of genes and proteins in the brain cells of psychopathic violent offenders.