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ISRIB, a tiny molecule identified by University of California, San Francisco (UCSF) researchers can repair the neural and cognitive effects of concussion in mice weeks after the damage, according to a new study.

ISRIB blocks the integrated stress response (ISR), a quality control process for protein production that, when activated chronically, can be harmful to cells.

The study, which was recently published in the Proceedings of the National Academy of Sciences, discovered that ISRIB reverses the effects of traumatic brain injury (TBI) on dendritic spines, an area of neurons vital to cognition. The drug-treated mice also showed sustained improvements in working memory.

A toddler is thriving after doctors in the U.S. and Canada used a novel technique to treat her before she was born for a rare genetic disease that caused the deaths of two of her sisters.

Ayla Bashir, a 16-month-old from Ottawa, Ontario, is the first child treated as a fetus for Pompe disease, an inherited and often fatal disorder in which the body fails to make some or all of a crucial protein.

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Gene therapies can scale economically, but not just with practices adapted from traditional biologics. According to Avantor, gene therapies pose unique material, workflow, and partnering challenges.

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For decades, scientists have sought to create red blood cells in the lab – a “holy grail” that some hoped could ease regional blood shortages, especially for people with rare blood types.

But now British researchers say they have overcome a major barrier that has plagued many scientists: creating enough red cells to fill a blood bag. Their findings are published in the journal Nature Communications.

Continue reading “Using stem cells to create an endless supply of blood” »

Scientists at the University of Pittsburgh School of Medicine have discovered the missing puzzle piece in the mystery of how melanoma tumors control their mortality.

In a paper published in Science this week, Jonathan Alder, Ph.D. and his team describe how they discovered the perfect combination of genetic alterations that tumors use to promote explosive growth and prevent their own demise, a development that could change the way oncologists understand and treat melanoma.

“We did something that was, in essence, obvious based on previous basic research and connected back to something that is happening in patients,” said Alder, assistant professor in the Division of Pulmonary, Allergy and Critical Care Medicine at Pitt’s School of Medicine.

Insilico Medicine is radiating heat amid the biotech winter, kindling its fires with a Sanofi collaboration that could be worth up to $1.2 billion in biobucks—the AI drug discovery company’s larges | Insilico Medicine is radiating heat amid the biotech winter, kindling its fires with a Sanofi collab that could be worth up to $1.2 billion in biobucks—the AI drug discovery company’s largest deal to date.

Recently, two patients in the United Kingdom received two small doses of lab-grown blood samples as part of the RESTORE trial.

Image Credit: Sashkin / Shutterstock.com

About the RESTORE trial

Continue reading “First transfusions of lab-grown blood” »

The findings come, in part, from nine patients involved in an ongoing Swiss study that is seeking to restore movement to people with paralysis.

All nine rapidly regained the ability to stand and walk with the help of implants that electrically stimulate spinal nerves that control lower-body movement.

Now the researchers are reporting that they’ve identified a specific group of cells in the lower spine that appear necessary for that movement recovery to happen.

Researchers at UC Riverside are paving the way for diabetes and cancer patients to forget needles and injections, and instead take pills to manage their conditions.

Some drugs for these diseases dissolve in water, so transporting them through the intestines, which receive what we drink and eat, is not feasible. As a result, these drugs cannot be administered by mouth. However, UCR scientists have created a chemical “tag” that can be added to these drugs, allowing them to enter blood circulation via the intestines.

The details of how they found the tag, and demonstrations of its effectiveness, are described in a new Journal of the American Chemical Society paper.