The main goal of our research is to develop
new therapies for Duchenne and Becker muscular dystrophy (DMD and BMD) and
other neuromuscular dystrophies which currently have no cure. DMD is the most
common muscular dystrophy in children. DMD patients suffer from
progressive muscle atrophy and weakness, lose independent ambulation by
the age of 13 years and often die in their third decade.
Our laboratory focuses on understanding
biochemical and molecular mechanisms leading to muscle dystrophy and the significant
processes contributing to its secondary effects and disease progression, such
as chronic inflammation and fibrosis. We are using diverse anti-inflammatory
and anti-fibrotic agents and also combination therapies, to tackle the massive
inflammation and fibrosis to improve outcomes in mouse models of DMD and of congenital
muscular dystrophy (CMD). We also combined different novel strategies of gene
therapy, small molecules and nanotechnology such as liposomes, exosomes and
other nanoparticles to deliver drugs specifically to muscles. Diverse
methodologies, including mouse models, human muscle biopsies, primary muscle
satellite cells, and bioinformatics techniques are employed. We use novel high
throughput sequencing (RNA-Seq) platform enabling us to identify novel genes
that promote muscle regeneration and seek to extend the animal findings to
humans. In addition to pure translational studies, biomarkers in our laboratory
are evaluated as an aid to study Duchenne and Becker muscular dystrophy patients'
physical activity and performance.
Figure 1. Model for the impaired regeneration mechanism in dy2J/dy2J mouse skeletal muscle (Yanay et al 2019).
Figure 2. Treatment with nano-liposomes (NSSL), remotely loaded with steroid decreased serum TGF-β and reduced diaphragm macrophage infiltration. In the long-term, NSSL-MPS demonstrated improved muscle strength and mobility compared to MPS as-is and induced lower tibia and lumbar vertebrae osteoporosis. (Turjeman and Yanay et al 2018)