In a groundbreaking development, scientists aboard the International Space Station (ISS) have successfully grown tiny human brains, or organoids, in space to aid in the search for treatments for neurological diseases such as Alzheimer’s, Parkinson’s, and spinal cord injuries.
This experiment could revolutionize research on these currently untreatable conditions by providing an accurate model for testing new treatments.
The low gravity environment of space played a crucial role in enabling the growth of human brain organoids in just 72 hours-an accomplishment that would typically take months on Earth.
These organoids, clusters of cells that replicate the structure and function of a human brain, allowed researchers to explore new methods of curing neurological diseases with remarkable speed and precision.
The experiment was led by Axonis Therapeutics, a biotechnology company that developed a promising new gene therapy using viral vectors to target and treat the central nervous system (CNS).
These viral vectors, which are modified viruses, could offer new hope for treating diseases such as Alzheimer’s, Parkinson’s, and spinal injuries by delivering beneficial gene therapies directly to CNS cells.
How does the treatment work?
Gene therapy has long been seen as a potential treatment for many neurological disorders.
Axonis’ treatment involves using viral vectors to introduce a fluorescent protein gene into neurons, which would cause them to glow neon green if successfully delivered.
Initial tests of the therapy on mice showed promising results, but the next crucial step was testing in an accurate human model. That’s where the ISS came in.
On Earth, CNS cells grow in two-dimensional layers due to gravity, making it challenging to replicate the complex, three-dimensional structure of brain cells.
In space, however, the lack of gravity allowed the cells to form three-dimensional brain organoids, creating a far more accurate representation of human brain tissue.
In August 2023, Axonis sent a cargo of frozen materials-including viral vectors, mature neurons, and astrocytes (support cells for neurons)-to the ISS.
The materials were injected into a specialized culturing system known as BioCell, where they were exposed to the viral vectors.
Researchers hoped the viral vectors would insert the fluorescent protein gene into the neurons, causing them to glow if successful.
In just 72 hours, the experiment yielded astounding results. The neurons formed into complex three-dimensional structures and began to glow, confirming that the gene therapy had been successfully delivered.
“We were able to both validate the gene therapy targeting ability and show that we rapidly formed 3D assembloid models of the human brain,” said Shane Hegarty, co-founder and chief scientific officer of Axonis Therapeutics.
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