Version 1
: Received: 30 April 2024 / Approved: 1 May 2024 / Online: 1 May 2024 (07:27:08 CEST)
How to cite:
Gandhi, S.; Sweeney, G.; Perry, C. G. Recent Advances in Pre-clinical Development of Adiponectin Receptor Agonist Therapies for Duchenne Muscular Dystrophy. Preprints2024, 2024050015. https://doi.org/10.20944/preprints202405.0015.v1
Gandhi, S.; Sweeney, G.; Perry, C. G. Recent Advances in Pre-clinical Development of Adiponectin Receptor Agonist Therapies for Duchenne Muscular Dystrophy. Preprints 2024, 2024050015. https://doi.org/10.20944/preprints202405.0015.v1
Gandhi, S.; Sweeney, G.; Perry, C. G. Recent Advances in Pre-clinical Development of Adiponectin Receptor Agonist Therapies for Duchenne Muscular Dystrophy. Preprints2024, 2024050015. https://doi.org/10.20944/preprints202405.0015.v1
APA Style
Gandhi, S., Sweeney, G., & Perry, C. G. (2024). Recent Advances in Pre-clinical Development of Adiponectin Receptor Agonist Therapies for Duchenne Muscular Dystrophy. Preprints. https://doi.org/10.20944/preprints202405.0015.v1
Chicago/Turabian Style
Gandhi, S., Gary Sweeney and Christopher G.R Perry. 2024 "Recent Advances in Pre-clinical Development of Adiponectin Receptor Agonist Therapies for Duchenne Muscular Dystrophy" Preprints. https://doi.org/10.20944/preprints202405.0015.v1
Abstract
Duchenne muscular dystrophy (DMD) is caused by genetic mutations in the cytoskeletal-sarcolemmal anchor protein dystrophin. Repeated cycles of sarcolemmal tearing and repair lead to a variety of secondary cellular and physiological stressors that are thought to contribute to weakness, atrophy, and fibrosis. Collectively, these stressors can contribute to a pro-inflammatory milieu in locomotor, cardiac, and respiratory muscles. Given the many unwanted side effects that accompany current anti-inflammatory steroid-based approaches for treating DMD (e.g., glucocorticoids), there is a need to develop new therapies that address inflammation and other cellular dysfunctions. Adiponectin receptor (AdipoR) agonists, which stimulate AdipoR1 and R2 isoforms on various cell types, have emerged as therapeutic candidates for DMD due to their anti-inflammatory, anti-fibrotic, and pro-myogenic properties in pre-clinical human and rodent DMD models. Although these molecules represent a new direction for therapeutic intervention, the mechanisms through which they elicit their beneficial effects are not yet fully understood, and DMD-specific data is limited. The overarching goal of this review is to investigate how adiponectin signaling may ameliorate pathology associated with dystrophin deficiency through inflammatory-dependent and -independent mechanisms, and to determine if current data supports their future progression to clinical trials.
Biology and Life Sciences, Biochemistry and Molecular Biology
Copyright:
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
