How to Manufacture Blood

Calendar February 13, 2023 | Posted by admin

Researchers in the U.K. have achieved something of a world first: they have manufactured blood in the lab, which they’ve since administered to humans. The clinical trial will aim to test the safety and effectiveness of the lab-made blood in at least 10 healthy people. Two volunteers have already received a dose.  The scientists — from the University of Cambridge, the National Health Service and the University of Bristol — are keen to find out whether their novel blood can last as long as normal red blood cells (which normally stay alive for about 120 days inside the human body) and whether there are any side effects.

Transfusing donated blood has saved countless lives, allowing patients to get through complicated operations in good health. Blood products also help to treat chronic conditions such as sickle cell anemia. But blood donation, as a system, has many drawbacks. It requires a complicated infrastructure to collect and deliver blood where it’s needed safely. That requires adequate refrigeration all along the route, and while that might be relatively straightforward for developed countries, it remains a challenge elsewhere in the world. Rarer blood types also suffer from dwindling supplies in the blood banks, which often means it’s harder to find a suitable blood match for certain racial and ethnic groups. It’s also costly to maintain the infrastructure; the average donation of less than half a liter of blood costs the U.K.’s National Health Service approximately £130 ($155). That’s why scientists from around the world, often funded by military agencies, have been searching for more practical alternatives for decades. Still, it’s an endeavor that has thus far enjoyed limited success.

“After 9/11, the U.S. Army invested millions of dollars in producing a blood replicant to be used for casualties in the battlefield, but it came to naught,” says Lt. Col. Matthew Armstrong, who studies the fluid dynamics of blood at the United States Military Academy at West Point.

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Crispr Can Edit Directly Genes Inside Human Bodies

Calendar September 30, 2022 | Posted by admin

A decade ago, biologists Jennifer Doudna and Emmanuelle Charpentier published a landmark paper describing a natural immune system found in bacteria and its potential as a tool for editing the genes of living organisms. A year later, in 2013, Feng Zhang and his colleagues at the Broad Institute of MIT and Harvard reported that they’d harnessed that system, known as Crispr, to edit human and animal cells in the lab. The work by both teams led to an explosion of interest in using Crispr to treat genetic diseases, as well as a 2020 Nobel Prize for Doudna and Charpentier.

Many diseases arise from gene mutations, so if Crispr could just snip out or replace an abnormal gene, it could in theory correct the disease. But one of the challenges of turning test tube Crispr discoveries into cures for patients has been figuring out how to get the gene-editing components to the place in the body that needs treatment.

One biotech company, Crispr Therapeutics, has gotten around that issue by editing patients’ cells outside the body. Scientists there have used the tool to treat dozens of people with sickle cell anemia and beta thalassemia—two common blood disorders. In those trials, investigators extract patients’ red blood cells, edit them to correct a disease-causing mutation, then infuse them back into the body.

But this “ex vivo” approach has downsides. It’s complex to administer, expensive, and has limited uses. Most diseases occur in cells and tissues that can’t be easily taken out of the body, treated, and put back in. So the next wave of Crispr research is focused on editing “in vivo”—that is, directly inside a patient’s body. Last year, Intellia Therapeutics was the first to demonstrate that this was possible for a disease called transthyretin amyloidosis. And last week, the Cambridge, Massachusetts-based biotech company showed in-the-body editing in a second disease.

Source: https://www.intelliatx.com/
AND
https://www.wired.com/

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