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

Abstract

Muscular dystrophies are a group of genetic muscular diseases characterized by impaired muscle regeneration, which leads to pathological inflammation that drives muscle wasting and eventually results in weakness, functional dependency, and premature death. The most known causes of death include respiratory muscle failure due to diaphragm muscle decay. There is no definitive treatment for muscular dystrophies, and conventional therapies aim to ameliorate muscle wasting by promoting physiological muscle regeneration and growth. However, their effects on muscle function remain limited, illustrating the requirement for major advancements in novel approaches to treatments, such as nanomedicine. Nanomedicine is a rapidly evolving field that seeks to optimize drug delivery to target tissues by merging pharmaceutical and biomedical sciences. However, the therapeutic potential of nanomedicine in muscular dystrophies is poorly understood. This review highlights recent work in the application of nanomedicine in treating muscular dystrophies. First, we discuss the history and applications of nanomedicine from a broader perspective. Second, we address the use of nanoparticles for drug delivery, gene regulation, and editing to target Duchenne muscular dystrophy and myotonic dystrophy. Next, we highlight the potential hindrances and limitations of using nanomedicine in the context of cell culture and animal models. Finally, the future perspectives for using nanomedicine in clinics are summarized with relevance to muscular dystrophies.

Deletion of Dux ameliorates muscular dystrophy in mdx mice by attenuating oxidative stress via Nrf2

“…Muscular dystrophy is a heterogeneous group of congenital neuromuscular disorders whose clinical signs include myalgia, skeletal muscle weakness, hypotonia, and atrophy, resulting in progressive muscle disability and loss of mobility 1 . Muscular dystrophy can also affect the heart and respiratory muscles and even shorten life expectancy 2,3 . Muscular dystrophy includes Duchenne muscular dystrophy (DMD), Becker muscular dystrophy, facioscapulohumeral muscular dystrophy (FSHD), and limb‐girdle muscular dystrophy, among others, of which DMD is the most common type 4 .…”

Nanodrug Delivery Systems for Myasthenia Gravis: Advances and Perspectives

“…The application of steroidal nano-drugs in Duchenne muscular dystrophy (DMD) can selectively target the dystrophic tissue and significantly reduce adverse effects following long-term treatment [119]. IL-4-and IL-10-conjugated gold nanoparticles can attenuate inflammatory muscle injury through immune regulation, which promotes Treg regeneration and improves muscle function [120]. The immunomodulatory camouflage nano-platform, which has an innovative structure, can effectively evade immune clearance and reduce autoimmune reactions during cancer treatment [121].…”

Non‐Viral Nucleic Acid Delivery System for RNA Therapeutics

“…[130] The combination of siRNA and cisplatin carried by poly(ethylene glycol)-block-poly(aminolated glycidyl methacrylate)-block-poly(2-(diisopropyl amino) ethyl methacrylate) (PEG-b-PAGA-b-PDPA) triblock copolymers was capable of inhibiting breast tumor growth and suppressing metastasis. [151] Poly (2-aminoethyl propylene phosphate) (PPE-EA) is a kind of cationic polymer based on polyphosphate, which is composed of main chain of phosphate and side chain of aminoethoxy group. [152] Plasmid DNA can be effectively compressed and embedded by PPE-EA, and can be continuously released with the degradation of polymer main chain, but the transfection efficiency is low because the PPA polymer has no obvious endosome buffer capacity.…”

“…[ 130 ] The combination of siRNA and cisplatin carried by poly(ethylene glycol)‐ block ‐poly(aminolated glycidyl methacrylate)‐ block ‐poly(2‐(diisopropyl amino) ethyl methacrylate) (PEG‐ b ‐PAGA‐ b ‐PDPA) triblock copolymers was capable of inhibiting breast tumor growth and suppressing metastasis. [ 151 ]…”