Lifeboat Foundation

In a paper published in the July 31 issue of Science Translational Medicine, researchers at Fred Hutchinson Cancer Research Center used CRISPR-Cas9 to edit long-lived blood stem cells to reverse the clinical symptoms observed with several blood disorders, including sickle cell disease and beta-thalassemia.

It’s the first time that scientists have specifically edited the genetic makeup of a specialized subset of adult blood stem cells that are the source of all cells in the blood and immune system.

The proof-of-principle study suggests that efficient modification of targeted stem cells could reduce the costs of gene-editing treatments for blood disorders and other diseases while decreasing the risks of unwanted effects that can occur with a less discriminating approach.

Mr. Epstein’s vision reflected his longstanding fascination with what has become known as transhumanism: the science of improving the human population through technologies like genetic engineering and artificial intelligence. Critics have likened transhumanism to a modern-day version of eugenics, the discredited field of improving the human race through controlled breeding.

Mr. Epstein, the accused sex trafficker, was fascinated by eugenics. He told scientists and others of his vision of using his New Mexico ranch to impregnate women.

The co-inventor of the groundbreaking gene editing technology talks to OneZero about a world where illness could be diagnosed in minutes.

But getting human cells to grow in another species is not easy. Nakauchi and colleagues announced at the 2018 American Association for the Advancement of Science meeting in Austin, Texas that they had put human iPS cells into sheep embryos that had been engineered not to produce a pancreas. But the hybrid embryos, grown for 28 days, contained very few human cells, and nothing resembling organs. This is probably because of the genetic distance between humans and sheep, says Nakauchi.

The research could eventually lead to new sources of organs for transplant, but ethical and technical hurdles need to be overcome.

Medicine has a “Goldilocks” problem. Many therapies are safe and effective only when administered at just the right time and in very precise doses – when given too early or too late, in too large or too small an amount, medicines can be ineffective or even harmful. But in many situations, doctors have no way of knowing when or how much to dispense.

Now, a team of bioengineers led by UC San Francisco’s Hana El-Samad, PhD, and the University of Washington’s David Baker, PhD, have devised a remarkable solution to this problem – “smart” cells that behave like tiny autonomous robots which, in the future, may be used to detect damage and disease, and deliver help at just the right time and in just the right amount.

Gene editing is advancing at a faster pace than most of us can keep up with. One significant recent announcement was gene editing tool CRISPR’s application to non-genetic diseases thanks to a new ability to edit single letters in RNA.

Even as CRISPR reaches milestones like this, scientists continue to find new uses for it to treat genetic conditions. The next one that will hit clinics is a CRISPR treatment for a form of blindness called Leber congenital amaurosis (LCA).

Having been approved by the FDA in December, the treatment will be the first of its kind to be trialed in the US.

Continue reading “First Human CRISPR Trial in the US Aims to Cure Inherited Blindness” »

A Sydney team has developed a box jellyfish antidote so simple it can go on as a spray. But it’s only the first step.