Review of Nanoparticle-Based Drug Delivery Methods in Conjunction With Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy
DOI:
https://doi.org/10.58445/rars.120Keywords:
Biotechnology, Medicine, Nanotechnology, Rare Diseases, Spinal Muscular Atrophy, Antisense Oligonucleotides, Drug-Delivery SystemsAbstract
Nanoparticles have shown great potential as drug delivery systems that are highly effective while also enhancing the chemical properties of the drug itself. In the last decade, numerous studies and clinical trials have shown that nanotechnology-based treatment strategies are significantly more effective and versatile in the field of biomedicine, especially when used as nanocarriers for treatments in rare neural, cardiovascular and blood disorders. Nanoparticle-based drug delivery systems are especially prominent solutions for neurological diseases because of their ability to cross the blood-brain barrier and blood-cerebrospinal fluid barrier, which block most endogenous and exogenous substances from entering into the central nervous system. Spinal Muscular Atrophy is a rare, genetic neurodegenerative disease affecting lower motor neurons and is the leading cause of genetic infant mortality. Spinal Muscular Atrophy has a prevalence rate of 1 in 6,000 in infants, and its severity in infants results in a high mortality rate, decreasing the average lifespan of patients to only 18 months. Antisense Oligonucleotides are a genetic treatment for SMA which function by split-slicing pre-messenger RNA and halting protein translation. Due to their poor penetration and low cellular uptake, they are ineffective when trying to mediate sites in the central nervous system. However, polymer, peptide and exosomes nanoparticles have shown to be promising nanocarriers which can enhance the drug’s chemical properties in offsetting the disease and allow it to reach into the central nervous system with increased target specification. In addition, these nanoparticles exhibit various features that enable them to directly overcome the limitations of Antisense Oligonucleotides. This review provides a discussion of advancement in nanotechnology-based drug delivery systems specified to amelioration of ASOs relevant to SMA disease progression and its usage in developing a critically-needed effective and accessible treatment for SMA patients.
References
Rezaei, Razieh. The Role of Nanomaterials in the Treatment of Diseases and Their Effects on the Immune System | Open Access Macedonian Journal of Medical Sciences. 16 June 2019, www.oamjms.eu/index.php/mjms/article/view/oamjms.2019.486. Accessed 31 Oct. 2022
Reichmuth, Oberli, Jaklenec, Langer, Blankschtein, Andreas, Matthias, Ana, Robert, Daniel. “mRNA Vaccine Delivery Using Lipid Nanoparticles.” Future Science, Apr. 2014, www.future-science.com/doi/10.4155/tde-2016-0006. Accessed 31 Oct. 2022.
“From Influenza to COVID-19: Lipid Nanoparticle mRNA Vaccines at the Frontiers of Infectious Diseases.” Acta Biomaterialia, vol. 131, Elsevier BV, Sept. 2021, pp. 16–40, doi:10.1016/j.actbio.2021.06.023.
Soni, Shringika et al. “Nanomedicine in Central Nervous System (CNS) Disorders: A Present and Future Prospective.” Advanced pharmaceutical bulletin vol. 6,3 (2016): 319-335. doi:10.15171/apb.2016.044. Accessed 31 Oct. 2022
Wang, G Y et al. “Advances in nanotechnology-based strategies for the treatments of amyotrophic lateral sclerosis.” Materials today. Bio vol. 6 100055. 4 May. 2020, doi:10.1016/j.mtbio.2020.100055. Accessed 31 Oct. 2022
Gloor, S M et al. “Molecular and cellular permeability control at the blood-brain barrier.” Brain research. Brain research reviews vol. 36,2-3 (2001): 258-64. doi:10.1016/s0165-0173(01)00102-3. Accessed 31 Oct. 2022
Ribovski, Laís et al. “Polymeric Nanoparticles Properties and Brain Delivery.” Pharmaceutics vol. 13,12 2045. 30 Nov. 2021, doi:10.3390/pharmaceutics13122045. Accessed 31 Oct. 2022
Abreu, Nicolas J, and Megan A Waldrop. “Overview of gene therapy in spinal muscular atrophy and Duchenne muscular dystrophy.” Pediatric pulmonology vol. 56,4 (2021): 710-720. doi:10.1002/ppul.25055. Accessed 31 Oct. 2022
Min, Hyun Su, et al. “Systemic Brain Delivery of Antisense Oligonucleotides Across the Blood–Brain Barrier With a Glucose‐Coated Polymeric Nanocarrier.” Angewandte Chemie International Edition, vol. 59, no. 21, Wiley, Mar. 2020, pp. 8173–80, doi:10.1002/anie.201914751. Accessed 10 Oct. 2022
Byun, Min Ji et al. “Advances in Nanoparticles for Effective Delivery of RNA Therapeutics.” Biochip journal vol. 16,2 (2022): 128-145. doi:10.1007/s13206-022-00052-5. Accessed 5 Oct. 2022
Shabanpoor, Fazel, et al. “Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy.” Nucleic Acid Therapeutics, vol. 27, no. 3, Mary Ann Liebert Inc, June 2017, pp. 130–43, doi:10.1089/nat.2016.0652. Accessed 10 Oct. 2022
Li, Qing. “Nusinersen as a Therapeutic Agent for Spinal Muscular Atrophy.” Yonsei medical journal vol. 61,4 (2020): 273-283. doi:10.3349/ymj.2020.61.4.273.Accessed 31 Oct. 2022.
Friedman, Adam D et al. “The smart targeting of nanoparticles.” Current pharmaceutical design vol. 19,35 (2013): 6315-29. doi:10.2174/13816128113199990375. Accessed 15 Oct. 2022
Tran, Phuc et al. “Delivery of Oligonucleotides: Efficiency with Lipid Conjugation and Clinical Outcome.” Pharmaceutics vol. 14,2 342. 1 Feb. 2022, doi:10.3390/pharmaceutics14020342. Accessed 31 Oct. 2022
Yang, Liu. “Efficient Delivery of Antisense Oligonucleotides Using Bioreducible Lipid Nanoparticles In Vitro and In Vivo.” Molecular Therapy - Nucleic Acids, 6 Mar. 2020, www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(20)30053-6. Accessed 27 Oct. 2022.
“First Antisense Drug Is Approved With Fleeting Success.” Nature, 14 Nov. 2019, www.nature.com/articles/d42859-019-00080-6?error=cookies_not_supported&code=9b66bf25-617f-4182-b789-e62e8445c5a8. Accessed 31 Oct. 2022.
Falzarano, Maria Sofia et al. “Nanoparticle delivery of antisense oligonucleotides and their application in the exon skipping strategy for Duchenne muscular dystrophy.” Nucleic acid therapeutics vol. 24,1 (2014): 87-100. doi:10.1089/nat.2013.0450. Accessed 25 Oct. 2022
Ugalmugle, Sumant, and Rupali Swain. “Rare Disease Treatment Market Size by Drug Type (Biologics, Non-biologics), by Therapeutic Area (Cancer, Blood-related Disorders, Central Nervous System (CNS), Respiratory Disorders, Musculoskeletal Disorders, Cardiovascular Disorders), by Patient (Adult, Pediatric), by Route of Administration (Oral, Injectable), Industry Analysis Report, Regional Outlook, Application Potential, Price Trends, Competitive Market Share and Forecast, 2020 – 2026.” Global Market Insights Inc., 12 Nov. 2020, www.gminsights.com/industry-analysis/rare-disease-treatment-market. Accessed 26 Oct. 2022.
Ghosh, Iman. “Infographic: Which Rare Diseases Are the Most Common?” Visual Capitalist, 12 Sept. 2019, www.visualcapitalist.com/which-rare-diseases-are-the-most-common. Accessed 26 Oct. 2022.
Spinal Muscular Atrophy Fact Sheet | National Institute of Neurological Disorders and Stroke. www.ninds.nih.gov/spinal-muscular-atrophy-fact-sheet. Accessed 27 Oct. 2022.
NORD - National Organization for Rare Disorders. “Spinal Muscular Atrophy.” NORD (National Organization for Rare Disorders), 12 Jan. 2022, www.rarediseases.org/rare-diseases/spinal-muscular-atrophy. Accessed 27 Oct. 2022.
Park, Hyun Bin et al. “Survival analysis of spinal muscular atrophy type I.” Korean journal of pediatrics vol. 53,11 (2010): 965-70. doi:10.3345/kjp.2010.53.11.965. Accessed 30 Oct. 2022
Montanez‑Gonzalez, Raquel. “A PCR-RFLP Method for Genotyping of Inversion 2Rc in Anopheles Coluzzii - Parasites and Vectors.” BioMed Central, 22 Mar. 2021, www.parasitesandvectors.biomedcentral.com/articles/10.1186/s13071-021-04657-x. Accessed 20 Oct. 2022.
NHS website. “Diagnosis.” nhs.uk, 18 Nov. 2021, www.nhs.uk/conditions/spinal-muscular-atrophy-sma/diagnosis. Accessed 27 Oct. 2022.
Brewer, Alex PharmD. “Zolgensma and Cost: What You Need to Know.” Healthline, 15 July 2022, www.healthline.com/health/drugs/zolgensma-cost. Accessed 31 Oct. 2022.
Liu, Angus. “Roche's Low-Price Evrysdi Will Take 'Meaningful' SMA Share from Biogen's Spinraza: Analyst.” Fierce Pharma, 11 Aug. 2020, https://www.fiercepharma.com/marketing/roche-s-low-price-evrysdi-will-take-meaningful-sma-share-from-biogen-s-spinraza-analyst.
Male, David, and Radka Gromnicova. “Nanocarriers for Delivery of Oligonucleotides to the CNS.” International journal of molecular sciences vol. 23,2 760. 11 Jan. 2022, doi:10.3390/ijms23020760. Accessed 10 Nov. 2022
Hou, Xucheng, et al. “Lipid Nanoparticles for Mrna Delivery.” Nature News, Nature Publishing Group, 10 Aug. 2021, https://www.nature.com/articles/s41578-021-00358-0. Accessed 10 Nov. 2022
Staton, Tracy. “Biogen's $375K Spinraza Price Puts a Sovaldi-Style Spotlight on Rare Disease Meds.” Fierce Pharma, 3 Jan. 2017, https://www.fiercepharma.com/pharma/biogen-s-375k-spinraza-price-puts-a-sovaldi-style-spotlight-rare-disease-meds. Accessed 15 Nov. 2022
Downloads
Posted
Categories
License
Copyright (c) 2023 Ansh Patel
This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License.