Organoids revolutionize approach to studying a variety of diseases

Organoids

Microscopes limit how well we can study cells. To understand complex processes, we must observe cells in conditions that mimic the body. Organoids have transformed this research.

Organoids and “mini-brains”

Organoids are three‑dimensional structures grown from stem cells that mimic real organs. Recent studies have used this method to explore a wide range of biological questions.

In one example, University of Cambridge researchers grew a “mini‑brain” from human stem cells. To test its function, they connected the organoid to a mouse spinal cord and nearby muscle. Remarkably, the mini‑brain sent electrical signals that made the muscles twitch. This model may help researchers study neurodegenerative diseases such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).

Spinal muscular atrophy

iPSC method

Speaking of SMA, researchers in Singapore have used organoids to uncover findings that may help people with the condition.

SMA is a neurodegenerative disease caused by a protein deficiency that leads to nerve loss, paralysis, and sometimes early death. Because it mainly affects children, the impact is especially severe. Scientists still know little about how SMA develops or how to treat it.

Researchers at A*STAR’s Institute of Molecular and Cell Biology (IMCB) addressed this gap by using the iPSC method to turn tissue samples from healthy individuals and people with SMA into spinal organoids.

They compared how the cells developed. Motor nerve cells from healthy donors fully formed by day 35, while cells from people with SMA began degenerating before reaching that stage.

They also discovered that the protein defect driving SMA forces motor nerve cells to divide, something they normally do not do. When the researchers blocked this abnormal division, they prevented the cells from dying.

CIRM funding

In Science and Technology Research News lead researcher Shi‑Yan Ng said this approach could also reveal clues about other diseases such as ALS.

“We are one of the first labs to report spinal organoid formation. Our study offers a new method for culturing human spinal‑cord‑like tissues that could be crucial for future research.”

Yesterday, the CIRM Board awarded almost $4 million to Ankasa Regenerative Therapeutics to develop a treatment for another serious back condition, degenerative spondylolisthesis.

Organoid modeling has also helped researchers study infectious disease. Scientists at the Max Planck Institute for Infection Biology in Berlin created fallopian tube organoids from normal human cells.. They used them to study chronic Chlamydia infections, a sexually transmitted infection. The team found that chronic infection causes lasting DNA changes as cells age. These changes persist even after the infection clears and may help explain the increased cervical cancer risk seen in women who have current or past Chlamydia infections.

New stem cell findings point to future therapies for spinal musculature atrophy

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