Nanomedicine, a valuable tool for skeletal muscle disorders: Challenges, promises, and limitations

Abstract

Muscular dystrophies are a group of rare genetic disorders characterized by progressive muscle weakness, which, in the most severe forms, leads to the patient's death due to cardiorespiratory problems. There is still no cure available for these diseases and significant effort is being placed into developing new strategies to either correct the genetic defect or to compensate muscle loss by stimulating skeletal muscle regeneration. However, the vast anatomical extension of the target tissue poses great challenges to these goals, highlighting the need for complementary strategies. Nanomedicine is an actively evolving field that merges nanotechnologies with biomedical and pharmaceutical sciences. It holds great potential in regenerative medicine, both in supporting tissue engineering and regeneration, and in optimizing drug and oligonucleotide delivery and gene therapy strategies. In this review, we will summarize the state-of-the-art in the field of nanomedicine applied to skeletal muscle regeneration. We will discuss the recent work toward the development of nanopatterned scaffolds for tissue engineering, the efforts in the synthesis of organic and inorganic nanoparticles for gene therapy and drug delivery applications, as well as their use as immune modulators. Although nanomedicine holds great promise for muscle and other degenerative diseases, many challenges still need to be systematically addressed to assure a smooth transition from the bench to the bedside.

Nano-Immunomodulation: A New Strategy for Skeletal Muscle Diseases and Aging?

“…Furthermore, the unique optical properties (fluorescence, plasmonic absorbance, etc.) of these NPs allow us to obtain precise spatiotemporal control (reviewed in [ 111 ]). However, despite these attractive properties, inorganic NPs are significantly less mature in terms of clinical translations and their potential toxicity is a significant matter of concern [ 112 ].…”

Nanomedicine for Treating Muscle Dystrophies: Opportunities, Challenges, and Future Perspectives

“…Muscular dystrophies are a group of genetic muscular diseases characterized by progressive muscle wasting and weakness that lead to functional dependency, disability, and premature death [ 1 ]. Altogether, muscular dystrophies involve mutations in over 40 genes with distinct pathogenic mechanisms and clinical presentations [ 2 ]. The global estimated prevalence of muscular dystrophies is 19.8—25.1 per 100,000 births [ 2 ].…”

“…Altogether, muscular dystrophies involve mutations in over 40 genes with distinct pathogenic mechanisms and clinical presentations [ 2 ]. The global estimated prevalence of muscular dystrophies is 19.8—25.1 per 100,000 births [ 2 ]. The two most common forms of muscular dystrophy are Duchenne muscular dystrophy (DMD) and myotonic dystrophy (DM) [ 1 ].…”

“…Over the last several years, nanomedicine has emerged as an attractive therapeutic option for many human diseases [ 9 ]. Nanoparticles have been successfully used to deliver oligonucleotides [ 10 ], cytokines [ 11 ], and other bioactive molecules, including growth factors [ 2 ], to promote the regeneration of target organs. Currently, several nanoparticles are being optimized to boost skeletal muscle mass and/or strength in different muscle-wasting conditions, including muscular dystrophies [ 2 , 8 ].…”

“…Nanoparticles have been successfully used to deliver oligonucleotides [ 10 ], cytokines [ 11 ], and other bioactive molecules, including growth factors [ 2 ], to promote the regeneration of target organs. Currently, several nanoparticles are being optimized to boost skeletal muscle mass and/or strength in different muscle-wasting conditions, including muscular dystrophies [ 2 , 8 ]. However, several barriers prevent the effective delivery of nanoparticles to dystrophic skeletal muscles.…”