GENE & CELL THERAPY
Our work pioneered the use of human artificial chromosomes and induced pluripotent stem (iPS) cells for muscle gene and cell therapies (Tedesco et al., Sci Transl Med 2011 and 2012; Maffioletti & Gerli et al., Nat Protoc 2015; Benedetti et al., EMBO Mol Med 2018). Current research projects are also investigating the use of small molecules to improve muscle stem cell delivery (Gerli & Moyle et al., Stem Cell Reports 2019)
We have reported the first isogenic, 3D human iPSC-derived, multilineage artificial skeletal muscle for complex disease modelling and beyond (Maffioletti & Sarcar, Cell Reports 2019). Current projects are focusing on extending this platform to tissue replacement, drug screening and therapy development for acute & chronic muscle/neuromuscular diseases.
We are adapting our gene/cell therapy and tissue engineering platforms to model severe and incurable neuromuscular diseases (Steele-Stallard HB et al., Front Physiol 2018). We started with skeletal muscle laminopathies and are currently investigating strategies to model several other neuromuscular disorders using iPSCs in 2D and 3D cultures.