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Non-viral delivery of nucleic acid for treatment of rare diseases of the muscle

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Abstract

Rare muscular disorders (RMDs) are disorders that affect a small percentage of the population. The disorders which are attributed to genetic mutations often manifest in the form of progressive weakness and atrophy of skeletal and heart muscles. RMDs includes disorders such as Duchenne muscular dystrophy (DMD), GNE myopathy, spinal muscular atrophy (SMA), limb girdle muscular dystrophy, and so on. Due to the infrequent occurrence of these disorders, development of therapeutic approaches elicits less attention compared with other more prevalent diseases. However, in recent times, improved understanding of pathogenesis has led to greater advances in developing therapeutic options to treat such diseases. Exon skipping, gene augmentation, and gene editing have taken the spotlight in drug development for rare neuromuscular disorders. The recent innovation in targeting and repairing mutations with the advent of CRISPR technology has in fact opened new possibilities in the development of gene therapy approaches for these disorders. Although these treatments show satisfactory therapeutic effects, the susceptibility to degradation, instability, and toxicity limits their application. So, an appropriate delivery vector is required for the delivery of these cargoes. Viral vectors are considered potential delivery systems for gene therapy; however, the associated concurrent immunogenic response and other limitations have paved the way for the applications of other non-viral systems like lipids, polymers, cell-penetrating peptides (CPPs), and other organic and inorganic materials. This review will focus on non-viral vectors for the delivery of therapeutic cargoes in order to treat muscular dystrophies.

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Acknowledgements

The authors would like to acknowledge the Indian Council of Medical Research (ICMR) for funding (GAP0246), the Council of Scientific & Industrial Research (CSIR), for the fellowship to DR, and the CSIR–Institute of Genomics and Integrative Biology for support. Figures were drawn using Canva (www.canva.com).

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Authors and Affiliations

  1. CSIR–Institute of Genomics and Integrative Biology, Mathura Road, New Delhi, 110025, India

    Divya Rao & Munia Ganguli

  2. Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, India

    Divya Rao & Munia Ganguli

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  1. Divya Rao
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  2. Munia Ganguli
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Correspondence to Munia Ganguli.

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Corresponding editor: Rakesh K Mishra

This article is part of the Topical Collection: The Rare Genetic Disease Research Landscape in India.

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Rao, D., Ganguli, M. Non-viral delivery of nucleic acid for treatment of rare diseases of the muscle. J Biosci 49, 27 (2024). https://doi.org/10.1007/s12038-023-00411-w

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