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The Miceli lab is focused around two main research topics: T cell biology and Duchenne Muscular Dystrophy (DMD). DMD is a lethal genetic disease of childhood, caused by mutations in DMD, which encodes the dystrophin protein. Without functional dystrophin, defects in sarcolemma stabilization lead to progressive muscle damage, while inherent stem cell defects limit efficiency of regeneration. Immune cells infiltrate muscle in response to damage, coordinating muscle satellite cell activation and muscle regeneration. In the face of the chronic damage in DMD, inflammation also drives fibrosis, promoting scarring and further limiting muscle regeneration. Progressive muscle weakness leads to loss of virtually all muscle function, respiratory and cardiac failure, and death between ages 20-30. The Miceli lab works closely with the CDMD clinic, clinical trials population and industry partners to use DMD clinic and clinical trial tissue sample to model human DMD, screen for new drugs/targets, assess therapeutic efficacy and mechanisms of action and immune consequences of dystrophin replacement and immune modulating therapies.
Because DMD patient biopsy tissue is of limited availability, we are developing and applying technology for robust assessment of single cell multi-parameter immune and muscle lineage cell surface marker and gene expression, including TCR/BCR cell V region usage from cryopreserved PBMC, and frozen muscle biopsy tissue. Additionally, we developed patient mutation specific DMD skeletal muscle and cardiac culture models. We have active collaborations with industry partners involved in dystrophin replacement/rescue and immune suppression in DMD, and access to remnant trial tissue and clinic samples for our studies. By examining the consequences of therapeutics on immunity, muscle regeneration, fibrosis and tissue tolerance in human DMD, we hope to identify therapeutic mechanism of action and efficacy, cell and molecular targets for drug discovery, and potential barriers to successful treatment. We hypothesize that these studies will identify novel immune and myogenic drivers of muscle regeneration, fibrosis and immune tolerance in human DMD.
The Miceli lab is focused around two main research topics: T cell biology and Duchenne Muscular Dystrophy (DMD). DMD is a lethal genetic disease of childhood, caused by mutations in DMD, which encodes the dystrophin protein. Without functional dystrophin, defects in sarcolemma stabilization lead to progressive muscle damage, while inherent stem cell defects limit efficiency of regeneration. Immune cells infiltrate muscle in response to damage, coordinating muscle satellite cell activation and muscle regeneration. In the face of the chronic damage in DMD, inflammation also drives fibrosis, promoting scarring and further limiting muscle regeneration. Progressive muscle weakness leads to loss of virtually all muscle function, respiratory and cardiac failure, and death between ages 20-30. The Miceli lab works closely with the CDMD clinic, clinical trials population and industry partners to use DMD clinic and clinical trial tissue sample to model human DMD, screen for new drugs/targets, assess therapeutic efficacy and mechanisms of action and immune consequences of dystrophin replacement and immune modulating therapies.
Because DMD patient biopsy tissue is of limited availability, we are developing and applying technology for robust assessment of single cell multi-parameter immune and muscle lineage cell surface marker and gene expression, including TCR/BCR cell V region usage from cryopreserved PBMC, and frozen muscle biopsy tissue. Additionally, we developed patient mutation specific DMD skeletal muscle and cardiac culture models. We have active collaborations with industry partners involved in dystrophin replacement/rescue and immune suppression in DMD, and access to remnant trial tissue and clinic samples for our studies. By examining the consequences of therapeutics on immunity, muscle regeneration, fibrosis and tissue tolerance in human DMD, we hope to identify therapeutic mechanism of action and efficacy, cell and molecular targets for drug discovery, and potential barriers to successful treatment. We hypothesize that these studies will identify novel immune and myogenic drivers of muscle regeneration, fibrosis and immune tolerance in human DMD.
研究兴趣
论文共 47 篇作者统计合作学者相似作者
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Shirley Nieves-Rodriguez,Florian Barthélémy,Jeremy D Woods,Emilie D Douine,Richard T Wang,Deirdre D Scripture-Adams, Kevin N Chesmore, Francesca Galasso,M Carrie Miceli,Stanley F Nelson
Frontiers in genetics (2023): 1216066
Molecular therapy. Nucleic acids (2018): 180-191
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