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Core technologies that already have been researched for our goal
- Prime Editing
- Prime editing is a next-generation gene-editing technology that enables precise and versatile correction of genetic mutations. Unlike traditional CRISPR methods that rely on cutting both strands of DNA, prime editing uses a modified Cas9 enzyme fused to a reverse transcriptase. Guided by a specialized RNA called pegRNA, this system can “search and replace” specific DNA sequences without causing double-strand breaks. This makes it a powerful and potentially safer tool for correcting disease-causing mutations—especially in the context of rare genetic disorders like LGMD2B.
- Adipose-derived Mesenchymal Stem Cell (AdMSC) Differentiation
- Adipose-derived mesenchymal stem cells (AdMSCs) are multipotent stem cells isolated from fat tissue. These cells have the unique ability to differentiate into various cell types, including muscle cells (myocytes), bone cells (osteoblasts), cartilage cells (chondrocytes), and fat cells (adipocytes). Through specific culture conditions and molecular signaling cues, AdMSCs can be directed to become specialized cell types, making them a promising tool for regenerative medicine and targeted therapies—especially in treating degenerative muscular diseases like LGMD2B.
- Muscle Stem Cell (MuSC)
- Muscle stem cells, also known as satellite cells, are a type of adult stem cell found in skeletal muscle tissue. They remain in a dormant (quiescent) state under normal conditions but become activated in response to muscle injury or stress. Once activated, MuSCs proliferate and differentiate into muscle fibers, playing a critical role in muscle regeneration and repair. Because of their inherent regenerative capacity, MuSCs are a key target in therapeutic research for muscle-wasting diseases such as LGMD2B. By harnessing and enhancing the function of MuSCs, scientists aim to restore muscle strength and function in patients with genetic muscle disorders.
- 3D Stem Cell Culture
- 3D stem cell culture is an advanced technique that allows stem cells to grow in a three-dimensional environment, more closely mimicking the natural conditions inside the human body. Unlike traditional 2D cultures, where cells grow in flat layers, 3D systems support more realistic cell-cell and cell-matrix interactions, leading to more physiologically relevant behavior. This approach enhances cell differentiation, viability, and functionality—making it especially valuable in regenerative medicine, drug screening, and disease modeling. In the context of muscular disorders like LGMD2B, 3D stem cell culture enables researchers to better study muscle tissue development and test therapeutic strategies in a more accurate biological context.
- Skeletal Muscle Organoids
- Skeletal muscle organoids are three-dimensional, miniaturized tissue models that replicate the structure and function of human skeletal muscle in vitro. Derived from stem cells, these organoids self-organize into muscle-like structures that exhibit key features of real muscle tissue, such as aligned muscle fibers, contractile ability, and responsiveness to stimuli. By providing a physiologically relevant model, skeletal muscle organoids are a powerful tool for studying muscle development, disease mechanisms (such as in LGMD2B), and evaluating potential therapies. They offer a bridge between traditional cell culture and animal models, enabling more accurate and human-specific research.
- Muscle Engraftment: A Critical Step in LGMD2B Treatment
- One of the most important stages in treating LGMD2B is ensuring that the edited gene is successfully delivered and engrafted into the affected muscle tissue. After infusion into the body, the genetically corrected cells must integrate into the muscle environment, differentiate into functional muscle fibers, and contribute to tissue regeneration. Without effective engraftment, the therapeutic benefits of gene editing cannot be fully realized. This step is essential for restoring muscle strength and halting disease progression.
