Team Titin, a non-profit organization, will fund $50,000 to support the project “Generation and characterization of skeletal muscle organoids from TTN patients’ iPSC for therapeutic screening”.
This program will be managed by Dr. Isabelle Richard, Head of the “Progressive Muscular Dystrophies” team in collaboration with Dr. Sonia Albini, Research Fellow in the same team and Dr. Edoardo Malfatti, Neurologist and Myopathologist at the University of Paris-Est.
“Titinopathies” are caused by a broad spectrum of muscle and/or heart disorders due to changes in the titin gene (TTN). This giant protein (34350 amino acids) plays a role in the stability and organization of the muscle cell. It also works as a molecular spring for passive elasticity of muscle.
It is currently unpredictable how the titin protein will be affected by a given genetic change and this might impact on a patient’s symptoms. Future therapeutic approaches also might be unpredictable.
One of the tracks identified would be to use organoids to test drug treatments. Organoids are three-dimensional, muscle-tissue cultures derived from patients’ blood cells, and are then programmed and differentiated into muscle.
In this study, Dr. Isabelle Richard and her team will examine the mutated titin proteins and their function an then use the organoids to test treatments. “We are excited to bring our knowledge and expertise to this project. First, this will allow us to model the muscle disease associated with the lack or deficiency of titin in vitro using organoids in muscle-tissue. These organoids will serve as a screening platform to detect and identify new therapies.” Dr. Sonia Albini said.
To access the press release of the Titin Team organization : Team Titin Funds $50,000 grant (titinmyopathy.com)