Announcing a significant milestone in our mission to find treatments for Nemaline Myopathy (NM)! As part of our latest round of research grants, A Foundation Building Strength (AFBS) is officially teaming up with DNA Nanobots, marking our first-ever research partnership with a private biotechnology company.
This collaboration represents a bold step forward in diversifying our research portfolio. By bridging the gap between non-profit advocacy and private-sector innovation, we’re expanding our tactical approaches to overcome the most difficult hurdles in genetic medicine.
Meeting the “Size Challenge” of NEB
Nemaline Myopathy is a complex disorder with several genetic origins. One of the most common forms of NM is caused by mutations in the NEB gene. The NEB gene is responsible for producing nebulin, an essential protein that acts as a structural “ruler” in muscle cells to ensure they contract and function properly.
The NEB gene presents a unique and significant challenge for researchers because of its extraordinary size. In the world of gene therapy– where a “gene” is a segment of DNA providing instructions for a protein– standard delivery methods often have capacity limits. Because the NEB gene is so large, it requires innovative “large-cargo” solutions to deliver healthy instructions to muscle cells effectively.
The Solution: DNA Nanotechnology
Enter DNA Nanobots. Founded in 2021 as an Ohio State University Spin-Out Company, DNA Nanobots is built on over a decade of pioneering research by Professor Carlos E. Castro. Their mission is to reshape the future of healthcare by using DNA itself as a structural building block for advanced medical devices.
Researchers Christopher Lucas, PhD, and Patrick Halley, MS, are developing a “DNA nanobot” specifically designed to solve the size problem associated with NEB.
In this project, titled “A Novel Targeted, Non-Viral Gene Delivery System to deliver Nebulin for Nemaline Myopathy,” the team will use proprietary DNA nanotechnology to build a delivery system where the DNA is the vehicle. This “NEB nanobot” is engineered to carry the full-length NEB gene directly to affected muscle cells.
Expanding Our Research Horizons
This non-viral approach offers several exciting advantages that complement our existing research efforts:
- High Cargo Capacity: It is specifically built to accommodate the massive NEB gene.
- Reduced Immune Response: Because the system doesn’t use a virus as its vehicle as in other gene therapy approaches, it’s designed to avoid provoking an immune response, potentially allowing for more flexible dosing.
- Cost-Effectiveness: This technology offers the potential for a less expensive manufacturing process, making genetic medicine more accessible.
The Roadmap for Discovery
This grant will fund a comprehensive three-part research project:
- In Vitro Testing: Building the “NEB nanobot” and testing its ability to enter and function within muscle cells in a laboratory setting.
- Safety Assessment (In Vivo): Testing the nanobots in animal models to ensure they are well-tolerated by the body.
- Therapeutic Effectiveness: Evaluating how well the NEB nanobot restores muscle function in animal models of NEB-related Nemaline Myopathy.
Why This Matters
If successful, this project will provide the necessary pre-clinical evidence for a new category of treatment for NEB-related NM. By adding DNA nanotechnology to our toolkit, AFBS is expanding our exploration of research avenues to bring relief to our community.
At AFBS, we believe that a multi-pronged approach is the fastest way to finding effective treatments for Nemaline Myopathy. By partnering with world-class innovators like DNA Nanobots, we are ensuring that the NM community remains at the forefront of the next generation of medicine.
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A Foundation Building Strength (AFBS) is dedicated to accelerating the development of treatments for Nemaline Myopathy. Our mission is to fund cutting-edge research to find effective treatments while providing resources and a strong community for families affected by NM. We are proud to be at the forefront of driving Nemaline Myopathy research breakthroughs forward through collaborative efforts with research teams all over the world.
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