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.
face_with_colon_three circa 2017.
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.
“When we kept (the cells) continually dividing for a year, we were quite excited,” said Jan Frayne, a biochemist at the University of Bristol and one of the study’s lead authors.
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
The RESTORE trial is a single-center, randomized, controlled, phase I cross-over trial that is a joint effort between the National Health Service (NHS) Blood and Transplant (NHSBT) and the University of Bristol. The aim of this trial is to determine whether blood cells manufactured from donor stem cells perform better than red blood cells (RBCs) obtained from the same donor in recipients.
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.
Video surveillance, now viruses.
Duke researchers have captured the first real-time footage of viruses on the move, right before they hijack a cell.
Learn more about the Welsher lab here: https://www.welsherlab.org/
To read the full paper: https://www.nature.com/articles/s41592-022-01672-3
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This past Monday (Nov. 7th) I was given a behind the scenes look at Dr. Alfonso Fasano’s neuromodulation clinic, one of the busiest of its kind in the world.
On his schedule for that day were about 25 patients, which is fairly typical for him. But these were not exactly 25 patients that a typical doctor might see in a given day. They were patients whom the Canadian medical system had funneled to him, individuals who not even most movement disorder specialists, let alone neurologists or general practitioners, could properly treat. For him and his team though, it was just another Monday.