Human iPSC NMJ models to study neuromuscular diseases
Official title: Human-iPSC derived neuromuscular junctions as a model for neuromuscular diseases.
Funded at Case Western Reserve University · NIH-11394226 · Data updated 2026-11-12
Scientists are building patient-friendly lab models using stem cells from humans to study how neuromuscular diseases like ALS affect the connection between nerves and muscles.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Case Western Reserve University NIH-funded |
| Lab location | 1 site (Cleveland, United States) |
| Project start | September 8, 2023 |
| Project end | July 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $390,007 |
| NIH project number | 5R33NS133212-03 |
| Project ID | NIH-11394226 on NIH RePORTER |
Who could benefit from this research
✓Could help
Not a patient trial; this is a laboratory model development project using human-derived cells.
✕Less likely to help
Patients will not participate in this lab study; no direct patient testing is involved.
What this research studies
This project uses human-induced pluripotent stem cells (iPSCs) to create neuromuscular junctions in the lab, aiming to better emulate how nerves communicate with muscles in diseases such as ALS. The team at Case Western Reserve University will develop and test these human NMJ models to capture functional connections between motor neurons and muscles. By focusing on a human-based system rather than only animal models, they hope to identify targets that translate more reliably to patients. The work addresses a gap where many promising treatments in animals fail in people, by pushing toward models that reflect human biology.
Why it matters
Potential benefit: If successful, the research could improve our understanding of NMJ failure in neuromuscular diseases and guide the development of therapies that better help nerves communicate with muscles.
How similar studies have gone: Other human iPSC NMJ models show promise, but translating results to patients remains challenging, making this an important step forward.
Where this research is happening
-
Case Western Reserve University
Cleveland, United States Active
Researchers
- Principal investigator: Miranda, Helen C — Case Western Reserve University
- Study coordinator: Miranda, Helen C
Related research
Other NIH-funded projects on the same topics.
-
Scalable non-viral gene delivery for a common muscular dystrophy
A patient-focused project tests a non-viral therapy to reduce harmful DMPK RNA in muscle because this could lessen symptoms of myotonic dystrophy type 1.
-
Antibody therapy to protect muscles in Duchenne and related diseases
A small drug company is developing antibodies that target a key protein (LTBP4) to calm harmful signals in muscles affected by Duchenne muscular dystrophy and similar conditions, with a plan to test safety and dosing in people soon.
-
Human nerve–muscle junctions grown from patient stem cells to model ALS and related neuromuscular diseases
They are growing human nerve-to-muscle connections from patient stem cells to help develop better treatments for people with ALS and similar neuromuscular conditions.
-
Creating a treatment for neuromuscular diseases using anti-LTBP4.
This study is looking at a new treatment that targets a protein called LTBP4 to help improve muscle function and slow down Duchenne Muscular Dystrophy (DMD), so that patients can feel better and do more in their daily lives.
-
Using human stem cells to study communication between nerves and muscles in neuromuscular diseases.
This study is exploring how special stem cells can help us create models of the connections between nerves and muscles, which are important for movement, to better understand diseases like ALS and find new ways to treat them.
-
Wearable monitoring of walking and arm movement in adults with neuromuscular or metabolic movement disorders
This project uses a wrist/ankle wearable to continuously monitor walking and upper-limb movement in ambulant adults with neuromuscular diseases or obesity to see if digital measures like stride speed track real-life motor function.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.