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The derivation of human embryonic stem cells (hESCs) and the stunning discovery that somatic cells can be reprogrammed into human induced pluripotent stem cells (hiPSCs) holds the promise to revolutionize biomedical research and regenerative medicine. In this Review, we focus on disorders of the central nervous system and explore how advances in human pluripotent stem cells (hPSCs) coincide with evolutions in genome engineering and genomic technologies to provide realistic opportunities to tackle some of the most devastating complex disorders.


Advances in stem cell biology are paving new paths toward their use in the clinic, especially toward understanding and treating neurological and neurodegenerative disease.

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Harnessing an antitumor immune response has been a fundamental strategy in cancer immunotherapy. For over a century, efforts have primarily focused on amplifying immune activation mechanisms that are employed by humans to eliminate invaders such as viruses and bacteria. This “immune enhancement” strategy often results in rare objective responses and frequent immune-related adverse events (irAEs). However, in the last decade, cancer immunotherapies targeting the B7-H1/PD-1 pathway (anti-PD therapy), have achieved higher objective response rates in patients with much fewer irAEs.


This Perspective discusses the concept of immune normalization and how its underlying principles may help to augment, as well as design, cancer immunotherapies.

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When researchers fed mosquitoes a drug used to treat people for obesity, the insects were less interested in hunting for their next human meal ticket. Karen Hopkin reports.

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Believe it or not, mosquitoes don’t bite out of spite. Female mosquitoes of the species Aedes aegypti need the nutrients present in your plasma to ensure the proper development of their eggs. And though their thirst may seem unquenchable, the ladies actually take time to savor your blood once they’ve sipped their fill.

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Cancer cells are, in some respects, impressive: They can grow relentlessly, sidestep the aging process by becoming immortal, and evade the immune system’s persistent attacks. But in the process of acquiring such superpowers, the cells must occasionally relinquish other, more mundane skills—including the ability to produce certain nutrients.

Researchers at The Rockefeller University now announce the discovery of a rare tumor type that is unable to synthesize cholesterol, a molecule without which can’t survive.

“These cells become dependent on taking up cholesterol from their environment, and we can use this dependency to design therapies that block cholesterol uptake,” says Kivanç Birsoy, the Chapman Perelman Assistant Professor, who reports the findings in Nature.

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Scientists at the University of Virginia School of Medicine have identified a potential explanation for the mysterious death of specific brain cells seen in Alzheimer’s, Parkinson’s and other neurodegenerative diseases.

The new research suggests that the may die because of naturally occurring in brain cells that were, until recently, assumed to be genetically identical. This variation – called “somatic mosaicism” – could explain why in the are the first to die in Alzheimer’s, for example, and why are the first to die in Parkinson’s.

“This has been a big open question in neuroscience, particularly in various neurodegenerative diseases,” said neuroscientist Michael McConnell of UVA’s Center for Brain Immunology and Glia, or BIG. “What is this selective vulnerability? What underlies it? And so now, with our work, the hypotheses moving forward are that it could be that different regions of the brain actually have a different garden of these [variations] in and that sets up different regions for decline later in life.”

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The so-called “zombie disease” has been reported in deer, elk, and moose across 24 US states, according to a new warning by the US Centers for Disease Control and Prevention (CDC).

As of January 2019, at least 251 counties across the US, from northern Montana to southern Texas, have reported CWD in free-ranging cervids, members of the deer family. Farther afield, there are similar concerns for reindeer in Norway, Finland, and, to a lesser extent, South Korea.

Scientifically known as chronic wasting disease (CWD), the contagious neurological disease gets its sensational nickname because of its effect on the brain of cervids, including North American elk or wapiti, red deer, mule deer, black-tailed deer, white-tailed deer, sika deer, reindeer, and moose. Deer that have been struck with the disease suffer from drastic weight loss, abnormal behavior, stumbling, drooling, lack of coordination, aggression, excessive thirst, and a fear of others.

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An experimental drug that bolsters ailing brain cells has raised hopes of a treatment for memory loss, poor decision making and other mental impairments that often strike in old age.

The drug could be taken as a daily pill by over-55s if clinical trials, which are expected to start within two years, show that the medicine is safe and effective at preventing memory lapses.

Tests in the lab showed that old animals had far better memory skills half an hour after receiving the drug. After two months on the treatment, brain cells which had shrunk in the animals had grown back, scientists found.

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