Lab-grown human brains could help cure world’s top killer disease

Daily Mail

Scientists have grown tiny human brains in space to research new treatments for Alzheimer’s, Parkinson’s and spinal injuries.
The low gravity environment on the International Space Station (ISS) enabled researchers to grow brain organoids in just 72 hours.
Axionis Therapeutics needed to perform tests using an accurate model of the human central nervous system, which is what prompted them to try and grow human brain organoids in space.
Within just 72 hours, the cells had not only organized themselves into three-dimensional human brain organoids, but were also glowing.
This study demonstrated that Axonis Therapeutics’ viral vector would be able to deliver gene therapy to human neurons.

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In order to investigate novel therapies for Parkinson’s disease, Alzheimer’s disease, and spinal injuries, researchers have cultured miniature human brains in space.

In just 72 hours, researchers were able to cultivate brain organoids on the International Space Station (ISS) thanks to its low gravity environment. On Earth, this process can take months.

By using artificially grown masses of cells that replicate the structure and function of a human organ, known as organoids, researchers were able to test a novel treatment approach for neurological disorders that are currently incurable.

Using a reprogrammed virus, the biotechnology company Axonis Therapeutics has created a promising new treatment that delivers beneficial gene therapy to cells in the central nervous system (CNS).

Viral vectors are modified viruses that may be especially helpful in treating spinal cord injuries, Parkinson’s disease, and Alzheimer’s disease.

Approximately 18,000 new spinal cord injuries occur in the United States each year, and nearly 600,000 Americans receive a diagnosis of either Parkinson’s or Alzheimer’s disease.

In mice, the company’s treatment proved effective. However, gene treatments that work well in rodent models frequently don’t work in people.

The attempt to cultivate human brain organoids in space was motivated by Axionis Therapeutics’ need to conduct tests using a precise model of the human central nervous system.

To investigate potential treatments for spinal injuries, Parkinson’s disease, and Alzheimer’s disease, researchers on the International Space Station (ISS) have created brain-like organoids in orbit.

It is challenging to cultivate the three-dimensional structure and complexity of brain cell functional groups because, although scientists can grow CNS cells on Earth, gravity forces them to organize into two-dimensional single layers.

The ISS researchers were able to test the efficacy of the new viral vector treatment because these space-grown organoids rapidly produced a very accurate model of the human brain.

“Normally, conducting research in space is not something you’d ever consider,” stated Shane Hegarty, Axonis Therapeutics’ co-founder and chief scientific officer.

“But by pushing the limits of science in a unique environment, the opportunity to leverage microgravity conditions can unlock a lot of untouched potential,” he added in a statement.

During a cargo resupply mission in August 2023, Axonis Therapeutics sent frozen vials of their viral vector, mature neurons, and astrocytes—cells that support and develop nerve cells—to the International Space Station.

These materials were then introduced into a device known as BioCell, a specialized cell-culturing system, by a team of researchers on the floating laboratory.

They inserted a fluorescent protein gene into the viral vector. The neurons would glow neon green if it was successful in entering them and introducing the gene.

In just 72 hours, the cells were glowing and had self-organized into three-dimensional human brain organoids.

Another study this year involved NASA astronaut Mike Barratt processing brain organoid samples aboard the ISS.

The ISS researchers grew the brain organelles and tested the viral vector’s efficacy using a specialized cell-culturing system called BioCell.

The day that the crew was performing the microscopy was crucial because, according to researcher Sheila Nielsen of BioServe Space Technologies, the company that developed BioCell, “we saw big aggregates of green, glowing 3D tissue.”.

“Shane and I were exchanging pictures of the organoids, and we were quite thrilled because this is something they had never seen on the ground,” she continued in the statement.

The ability of viruses to enter cells and introduce genetic material into them is highly effective.

By causing cell damage and facilitating the virus’s reproduction, the inserted genetic material makes their hosts ill and triggers an immune reaction.

In order to provide a targeted treatment for illness or injury, scientists can rewire viruses to deliver advantageous gene therapies to cells.

The ability of Axonis Therapeutics’ viral vector to deliver gene therapy to human neurons was shown in this study.

We were able to confirm the gene therapy targeting capability and demonstrate that we could quickly create 3D assembled models of the human brain, which was really exciting, Hegarty said.

The main innovation is that, in microgravity, mature neurons and astrocytes can quickly self-assemble into a brain model, something that is impossible on Earth. “.”.

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