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Biomacromolecule-mediated pulmonary delivery of siRNA and anti-sense oligos: challenges and possible solutions

Published online by Cambridge University Press:  15 December 2021

Yaseen Hussain Open the ORCID record for Yaseen Hussain [Opens in a new window] ,
Jing-Hao Cui ,
Haroon Khan ,
Pooyan Makvandi  and
Waqas Alam
Yaseen Hussain
Affiliation:
College of Pharmaceutical Sciences, Soochow University, Suzhou, Jiangsu215123, China
Jing-Hao Cui*
Affiliation:
College of Pharmaceutical Sciences, Soochow University, Suzhou, Jiangsu215123, China
Haroon Khan*
Affiliation:
Department of Pharmacy, Abdul Wali Khan University, Mardan23200, Pakistan
Pooyan Makvandi
Affiliation:
Istituto Italiano di Tecnologia, Centre for Micro-BioRobotics, viale Rinaldo Piaggio 34, 56025Pontedera, Pisa, Italy
Waqas Alam
Affiliation:
Department of Pharmacy, Abdul Wali Khan University, Mardan23200, Pakistan
*
Author for correspondence: Jing-Hao Cui, E-mail: [email protected]; Haroon Khan, E-mail: [email protected]
Author for correspondence: Jing-Hao Cui, E-mail: [email protected]; Haroon Khan, E-mail: [email protected]
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Abstract

Biomacromolecules have gained much attention as biomedicine carriers in recent years due to their remarkable biophysical and biochemical properties including sustainability, non-toxicity, biocompatibility, biodegradability, long systemic circulation time and ability to target. Recent developments in a variety of biological functions of biomacromolecules and progress in the study of biological drug carriers suggest that these carriers may have advantages over carriers of synthetic materials in terms of half-life, durability, protection and manufacturing facility. Despite the full pledge advancements in the applications of biomacromolecules, its clinical use is hindered by certain factors that allow the pre-mature release of loaded cargos before reaching the target site. The delivery therapeutics are degraded by systemic nucleases, cleared by reticulo-endothelial system, cleared by pulmonary mucus cilia or engulfed by lysosome during cellular uptake that has led to the failure of clinical therapy. It clearly indicates that there is a wide range of gaps in the results of experimental work and clinical applications of biomacromolecules. This review focuses mainly on the barriers (intracellular/extracellular) and hurdles to the delivery of biomacromolecules with special emphasis on siRNA as well as the delivery of antisense oligos in multiple pulmonary diseases, particularly focusing on lung cancer. Also, the challenges posed to such delivery and possible solutions have been highlighted.

Keywords

Lung cancerpulmonary delivery challengespulmonary diseasessiRNA/anti-sense oligos
Type
Review
Information
Expert Reviews in Molecular Medicine , Volume 23 , 2021 , e22
Copyright
Copyright © The Author(s), 2021. Published by Cambridge University Press

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References

Zhang, Y, Sun, T and Jiang, C (2018) Biomacromolecules as carriers in drug delivery and tissue engineering. Acta Pharmaceutica Sinica B 8, 34–50.CrossRefGoogle ScholarPubMed
FAO, F (2017) The Future of Food and Agriculture – Trends and Challenges. Annual Report.Google Scholar
Hanna, TP et al. (2020) Mortality due to cancer treatment delay: systematic review and meta-analysis. British Medical Journal 371, 371.Google ScholarPubMed
Kanwal, M, Ding, XJ and Cao, Y (2017) Familial risk for lung cancer. Oncology Letters 13, 535–542.CrossRefGoogle ScholarPubMed
W.H.O., Cancer 2018.Google Scholar
Manchado, E et al. (2016) A combinatorial strategy for treating KRAS-mutant lung cancer. Nature 534, 647–651.CrossRefGoogle ScholarPubMed
Shimomura, I et al. (2019) Drug library screen reveals benzimidazole derivatives as selective cytotoxic agents for KRAS-mutant lung cancer. Cancer Letters 451, 11–22.CrossRefGoogle ScholarPubMed
Sheu, R et al. (2020) Human transport of third hand tobacco smoke: a prominent source of hazardous air pollutants into indoor nonsmoking environments. Science Advances 6, eaay4109.CrossRefGoogle Scholar
Postmus, P et al. (2017) Early and locally advanced non-small-cell lung cancer (NSCLC): ESMO clinical practice guidelines for diagnosis, treatment and follow-up. Annals of Oncology 28, iv1–iv21.CrossRefGoogle ScholarPubMed
Chen, H et al. (2018) Stereotactic ablative radiation therapy versus surgery in early lung cancer: a meta-analysis of propensity score studies. International Journal of Radiation Oncology, Biology, Physics 101, 186–194.CrossRefGoogle ScholarPubMed
Usmani, OS (2019) Choosing the right inhaler for your asthma or COPD patient. Therapeutics and Clinical Risk Management 15, 461.CrossRefGoogle ScholarPubMed
McKelvey, MC et al. (2020) Targeting proteases in cystic fibrosis lung disease. Paradigms, progress, and potential. American Journal of Respiratory and Critical Care Medicine 201, 141–147.CrossRefGoogle ScholarPubMed
Yuan, S et al. (2015) Oxidation increases mucin polymer cross-links to stiffen airway mucus gels. Science Translational Medicine 7, 276ra27.CrossRefGoogle ScholarPubMed
Gon, Y and Hashimoto, S (2018) Role of airway epithelial barrier dysfunction in pathogenesis of asthma. Allergology International 67, 12–17.CrossRefGoogle ScholarPubMed
Chen, J et al. (2018) Nucleic acid-based therapeutics for pulmonary diseases. AAPS PharmSciTech 19, 3670–3680.CrossRefGoogle ScholarPubMed
Koli, U et al. (2014) SiRNA-based therapies for pulmonary diseases. Journal of Biomedical Nanotechnology 10, 1953–1997.CrossRefGoogle ScholarPubMed
de Souza Carvalho, C, Daum, N and Lehr, C-M (2014) Carrier interactions with the biological barriers of the lung: advanced in vitro models and challenges for pulmonary drug delivery. Advanced Drug Delivery Reviews 75, 129–140.CrossRefGoogle ScholarPubMed
Dua, K et al. (2019) The potential of siRNA based drug delivery in respiratory disorders: recent advances and progress. Drug Development Research 80, 714–730.CrossRefGoogle ScholarPubMed
Durymanov, M and Reineke, J (2018) Non-viral delivery of nucleic acids: insight into mechanisms of overcoming intracellular barriers. Frontiers in Pharmacology 9, 971.CrossRefGoogle ScholarPubMed
Dammes, N and Peer, D (2020) Paving the road for RNA therapeutics. Trends in Pharmacological Sciences 41, 755–775.CrossRefGoogle ScholarPubMed
Pei, D and Buyanova, M (2018) Overcoming endosomal entrapment in drug delivery. Bioconjugate Chemistry 30, 273–283.CrossRefGoogle ScholarPubMed
Dominska, M and Dykxhoorn, DM (2010) Breaking down the barriers: siRNA delivery and endosome escape. Journal of Cell Science 123, 1183–1189.CrossRefGoogle ScholarPubMed
Gao, Y et al. (2019) pH/Redox dual-responsive polyplex with effective endosomal escape for codelivery of siRNA and doxorubicin against drug-resistant cancer cells. ACS Applied Materials & Interfaces 11, 16296–16310.CrossRefGoogle ScholarPubMed
Large, DE et al. (2019) Advances in receptor-mediated, tumor-targeted drug delivery. Advanced Therapeutics 2, 1800091.CrossRefGoogle Scholar
Gazdar, AF, Bunn, PA and Minna, JD (2017) Small-cell lung cancer: what we know, what we need to know and the path forward. Nature Reviews Cancer 17, 725.CrossRefGoogle ScholarPubMed
Sadhasivam, J and Sugumaran, A (2020) Magnetic nanocarriers: emerging tool for the effective targeted treatment of lung cancer. Journal of Drug Delivery Science and Technology 55, 101493.CrossRefGoogle Scholar
Oun, R, Moussa, YE and Wheate, NJ (2018) The side effects of platinum-based chemotherapy drugs: a review for chemists. Dalton Transactions 47, 6645–6653.CrossRefGoogle ScholarPubMed
Itani, R and Al Faraj, A (2019) siRNA conjugated nanoparticles – a next generation strategy to treat lung cancer. International Journal of Molecular Sciences 20, 6088.CrossRefGoogle ScholarPubMed
Fehrenbach, H, Wagner, C and Wegmann, M (2017) Airway remodeling in asthma: what really matters. Cell and Tissue Research 367, 551–569.CrossRefGoogle ScholarPubMed
Kim, N et al. (2016) Barriers to inhaled gene therapy of obstructive lung diseases: a review. Journal of Controlled Release 240, 465–488.CrossRefGoogle ScholarPubMed
Cooney, AL, McCray, PB and Sinn, PL (2018) Cystic fibrosis gene therapy: looking back, looking forward. Genes 9, 538.CrossRefGoogle ScholarPubMed
Bardoliwala, D et al. (2019) Nanocarriers in effective pulmonary delivery of siRNA: current approaches and challenges. Therapeutic Delivery 10, 311–332.CrossRefGoogle ScholarPubMed
Chow, MY, Qiu, Y and Lam, JK (2020) Inhaled RNA therapy: from promise to reality. Trends in Pharmacological Sciences 41, 715–729.CrossRefGoogle Scholar
Dong, Y, Siegwart, DJ and Anderson, DG (2019) Strategies, design, and chemistry in siRNA delivery systems. Advanced Drug Delivery Reviews 144, 133–147.CrossRefGoogle ScholarPubMed
Liao, W et al. (2017) Oligonucleotide therapy for obstructive and restrictive respiratory diseases. Molecules 22, 139.CrossRefGoogle ScholarPubMed
Lee, SH et al. (2016) Current preclinical small interfering RNA (siRNA)-based conjugate systems for RNA therapeutics. Advanced Drug Delivery Reviews 104, 78–92.CrossRefGoogle ScholarPubMed
Chernikov, IV et al. (2017) Cholesterol-containing nuclease-resistant siRNA accumulates in tumors in a carrier-free mode and silences MDR1 gene. Molecular Therapy-Nucleic Acids 6, 209–220.CrossRefGoogle Scholar
Degors, IM et al. (2019) Carriers break barriers in drug delivery: endocytosis and endosomal escape of gene delivery vectors. Accounts of Chemical Research 52, 1750–1760.CrossRefGoogle ScholarPubMed
Lee, SJ et al. (2016) Delivery strategies and potential targets for siRNA in major cancer types. Advanced Drug Delivery Reviews 104, 2–15.CrossRefGoogle ScholarPubMed
Jung, J et al. (2012) Polymeric nanoparticles containing taxanes enhance chemoradiotherapeutic efficacy in non-small cell lung cancer. International Journal of Radiation Oncology, Biology, Physics 84, e77–e83.CrossRefGoogle ScholarPubMed
Miao, C et al. (2018) MicroRNAs in idiopathic pulmonary fibrosis, new research progress and their pathophysiological implication. Experimental Lung Research 44, 178–190.CrossRefGoogle ScholarPubMed
Chang, C-J et al. (2021) Overcoming interferon (IFN)-γ resistance ameliorates transforming growth factor (TGF)-β-mediated lung fibroblast-to-myofibroblast transition and bleomycin-induced pulmonary fibrosis. Biochemical Pharmacology 183, 114356.CrossRefGoogle ScholarPubMed
Su, Q, Sena-Esteves, M and Gao, G (2020) Production of recombinant adeno-associated viruses (rAAVs) by transient transfection. Cold Spring Harbor Protocols 2020, pdb. prot095596.CrossRefGoogle ScholarPubMed
Barba, AA et al. (2019) Lipid delivery systems for nucleic-acid-based-drugs: from production to clinical applications. Pharmaceutics 11, 360.CrossRefGoogle ScholarPubMed
Yan, Y et al. (2017) Aerosol delivery of stabilized polyester-siRNA nanoparticles to silence gene expression in orthotopic lung tumors. Biomaterials 118, 84–93.CrossRefGoogle ScholarPubMed
Gomes dos Reis, L et al. (2017) Inhaled gene delivery: a formulation and delivery approach. Expert Opinion on Drug Delivery 14, 319–330.CrossRefGoogle ScholarPubMed
Patil, S et al. (2019) The development of functional non-viral vectors for gene delivery. International Journal of Molecular Sciences 20, 5491.CrossRefGoogle ScholarPubMed
Mohammadinejad, R et al. (2020) In vivo gene delivery mediated by non-viral vectors for cancer therapy. Journal of Controlled Release 325, 249–275.CrossRefGoogle ScholarPubMed
d'Angelo, I et al. (2018) Hybrid lipid/polymer nanoparticles for pulmonary delivery of siRNA: development and fate upon in vitro deposition on the human epithelial airway barrier. Journal of Aerosol Medicine and Pulmonary Drug Delivery 31, 170–181.CrossRefGoogle ScholarPubMed
De Backer, L et al. (2015) Hybrid pulmonary surfactant-coated nanogels mediate efficient in vivo delivery of siRNA to murine alveolar macrophages. Journal of Controlled Release 217, 53–63.CrossRefGoogle ScholarPubMed
Ivanova, N et al. (2018) Silver nanoparticles as multi-functional drug delivery systems, in Nanomedicines. IntechOpen.CrossRefGoogle Scholar
Parhi, P, Mohanty, C and Sahoo, SK (2012) Nanotechnology-based combinational drug delivery: an emerging approach for cancer therapy. Drug Discovery Today 17, 1044–1052.CrossRefGoogle ScholarPubMed
Patra, JK et al. (2018) Nano based drug delivery systems: recent developments and future prospects. Journal of Nanobiotechnology 16, 71.CrossRefGoogle ScholarPubMed
Kusumoto, K et al. (2013) Lipid envelope-type nanoparticle incorporating a multifunctional peptide for systemic siRNA delivery to the pulmonary endothelium. ACS Nano 7, 7534–7541.CrossRefGoogle ScholarPubMed
De Carvalho Vicentini, FTM et al. (2013) Delivery systems and local administration routes for therapeutic siRNA. Pharmaceutical Research 30, 915–931.CrossRefGoogle Scholar
Lam, JK-W, Liang, W and Chan, H-K (2012) Pulmonary delivery of therapeutic siRNA. Advanced Drug Delivery Reviews 64, 1–15.CrossRefGoogle ScholarPubMed
Ni, S et al. (2018) GABAB receptor ligand-directed trimethyl chitosan/tripolyphosphate nanoparticles and their pMDI formulation for surviving siRNA pulmonary delivery. Carbohydrate Polymers 179, 135–144.CrossRefGoogle Scholar
Chow, MYT and Lam, JKW (2015) Dry powder formulation of plasmid DNA and siRNA for inhalation. Current Pharmaceutical Design 21, 3854–3866.CrossRefGoogle ScholarPubMed
Allen, TM and Cullis, PR (2004) Drug delivery systems: entering the mainstream. Science 303, 1818–1822.CrossRefGoogle Scholar
Liu, M et al. (2020) Recent advances in the development of nanoparticles for multimodality imaging and therapy of cancer. Medicinal Research Reviews 40, 909–930.CrossRefGoogle ScholarPubMed
Ansari, MT et al. (2020) Lipid-based nanocarriers for cancer and tumor treatment. Current Pharmaceutical Design 26, 4272–4276.CrossRefGoogle ScholarPubMed
Narenji, M, Talaee, M and Moghimi, H (2016) Investigating the effects of size, charge, viscosity and bilayer flexibility on liposomal delivery under convective flow. International Journal of Pharmaceutics 513, 88–96.CrossRefGoogle ScholarPubMed
Chuang, S-Y et al. (2018) Lipid-based nanoparticles as a potential delivery approach in the treatment of rheumatoid arthritis. Nanomaterials 8, 42.CrossRefGoogle ScholarPubMed
Chen, C, Ng, DYW and Weil, T (2020) Polymer bioconjugates: modern design concepts toward precision hybrid materials. Progress in Polymer Science 105, 101241.CrossRefGoogle Scholar
Liarou, E et al. (2018) Smart polymersomes and hydrogels from polypeptide-based polymer systems through α-amino acid N-carboxyanhydride ring-opening polymerization. From chemistry to biomedical applications. Progress in Polymer Science 83, 28–78.CrossRefGoogle Scholar
Indoria, S, Singh, V and Hsieh, MF (2020) Recent advances in theranostic polymeric nanoparticles for cancer treatment: a review. International Journal of Pharmaceutics 582, 119314.CrossRefGoogle ScholarPubMed
Shukla, R et al. (2019) Conclusion and Future Prospective of Polymeric Nanoparticles for Cancer Therapy, in Polymeric Nanoparticles as a Promising Tool for Anti-cancer Therapeutics. Amsterdam for science direct: Elsevier, pp. 389–408.CrossRefGoogle Scholar
Kim, J et al. (2020) Lung-targeted delivery of TGF-β antisense oligonucleotides to treat pulmonary fibrosis. Journal of Controlled Release 322, 108–121.CrossRefGoogle ScholarPubMed
Bai, D et al. (2019) Pulmonary delivery of NLRP3 antisense oligonucleotides are effective at preventing but not reversing established bleomycin-induced pulmonary fibrosis in mice, in C34. ILD AND IPF: CLINICAL AND MECHANISTIC STUDIES. American Thoracic Society. p. A4605–A4605.CrossRefGoogle Scholar
Carrer, M et al. (2020) Antisense oligonucleotides targeting Jagged 1 reduce house dust mite–induced goblet cell metaplasia in the adult murine lung. American Journal of Respiratory Cell and Molecular Biology 63, 46–56.CrossRefGoogle ScholarPubMed
Glassman, PM et al. (2020) Targeting drug delivery in the vascular system: focus on endothelium. Advanced Drug Delivery Reviews 157, 96–117.CrossRefGoogle ScholarPubMed
Yoon, Y-S et al. (2016) Macrophages programmed by apoptotic cells inhibit epithelial-mesenchymal transition in lung alveolar epithelial cells via PGE 2, PGD 2, and HGF. Scientific Reports 6, 1–18.CrossRefGoogle Scholar
Uemura, Y and Kobayashi, K (2019) Targeting murine alveolar macrophages by the intratracheal administration of locked nucleic acid containing antisense oligonucleotides. Drug Delivery 26, 803–811.CrossRefGoogle ScholarPubMed
Ubale, RV et al. (2018) Pulmonary administration of microparticulate antisense oligonucleotide (ASO) for the treatment of lung inflammation. AAPS PharmSciTech 19, 1908–1919.CrossRefGoogle ScholarPubMed
Vij, N (2011) Nano-based theranostics for chronic obstructive lung diseases: challenges and therapeutic potential. Expert Opinion on Drug Delivery 8, 1105–1109.CrossRefGoogle ScholarPubMed
Kolonko, AK et al. (2020) Chitosan nanocomplexes for the delivery of ENaC antisense oligonucleotides to airway epithelial cells. Biomolecules 10, 553.CrossRefGoogle ScholarPubMed
Martinovich, KM et al. (2018) The potential of antisense oligonucleotide therapies for inherited childhood lung diseases. Molecular and Cellular Pediatrics 5, 1–10.CrossRefGoogle ScholarPubMed
Guk, K et al. (2013) Acid-cleavable ketal containing poly (β-amino ester) for enhanced siRNA delivery. International Journal of Pharmaceutics 453, 541–550.CrossRefGoogle ScholarPubMed
Patil, ML et al. (2009) Internally cationic polyamidoamine PAMAM-OH dendrimers for siRNA delivery: effect of the degree of quaternization and cancer targeting. Biomacromolecules 10, 258–266.CrossRefGoogle ScholarPubMed
Mukhtar, M et al. (2020) Drug delivery to macrophages: a review of nano-therapeutics targeted approach for inflammatory disorders and cancer. Expert Opinion on Drug Delivery 17, 1239–1257.CrossRefGoogle ScholarPubMed
Vermeulen, LM et al. (2018) Methodologies to investigate intracellular barriers for nucleic acid delivery in non-viral gene therapy. Nano Today 21, 74–90.CrossRefGoogle Scholar
Wang, H and Mooney, DJ (2018) Biomaterial-assisted targeted modulation of immune cells in cancer treatment. Nature Materials 17, 761–772.CrossRefGoogle ScholarPubMed
Baharifar, H et al. (2020) Preparation of PEG-grafted chitosan/streptokinase nanoparticles to improve biological half-life and reduce immunogenicity of the enzyme. International Journal of Biological Macromolecules 143, 181–189.CrossRefGoogle ScholarPubMed
Ni, R, Feng, R and Chau, Y (2019) Synthetic approaches for nucleic acid delivery: choosing the right carriers. Life 9, 59.CrossRefGoogle ScholarPubMed
Sawada, S-I et al. (2020) Nanogel hybrid assembly for exosome intracellular delivery: effects on endocytosis and fusion by exosome surface polymer engineering. Biomaterials Science 8, 619–630.CrossRefGoogle ScholarPubMed
Ahmadzada, T, Reid, G and McKenzie, DR (2018) Fundamentals of siRNA and miRNA therapeutics and a review of targeted nanoparticle delivery systems in breast cancer. Biophysical Reviews 10, 69–86.CrossRefGoogle Scholar
Saw, PE and Song, E-W (2020) siRNA therapeutics: a clinical reality. Science China Life Sciences 63, 485–500.CrossRefGoogle ScholarPubMed
Hong, S-J et al. (2018) Targeted delivery of siRNA therapeutics using ligand mediated biodegradable polymeric nanocarriers. Current Pharmaceutical Design 24, 1788–1800.Google Scholar
Keil, TW, Baldassi, D and Merkel, OM (2020) T-cell targeted pulmonary siRNA delivery for the treatment of asthma. Wiley Interdisciplinary Reviews: Nanomedicine and Nanobiotechnology 12, e1634.Google ScholarPubMed
Ercole, F et al. (2015) Cholesterol modified self-assemblies and their application to nanomedicine. Biomacromolecules 16, 1886–1914.CrossRefGoogle ScholarPubMed
Bansal, A (2019) Non-viral vectors for gene delivery. Nanoscience & Nanotechnology-Asia 9, 4–11.CrossRefGoogle Scholar
Stewart, MP et al. (2016) Challenges in carrier-mediated intracellular delivery: moving beyond endosomal barriers. Wiley Interdisciplinary Reviews: Nanomedicine and Nanobiotechnology 8, 465–478.Google ScholarPubMed
Michlewska, S et al. (2018) Ruthenium dendrimers as carriers for anticancer siRNA. Journal of Inorganic Biochemistry 181, 18–27.CrossRefGoogle ScholarPubMed
He, Y et al. (2019) Rational particle design to overcome pulmonary barriers for obstructive lung diseases therapy. Journal of Controlled Release 314, 48–61.CrossRefGoogle ScholarPubMed
Osman, G et al. (2018) PEGylated enhanced cell penetrating peptide nanoparticles for lung gene therapy. Journal of Controlled Release 285, 35–45.CrossRefGoogle ScholarPubMed
Ge, C et al. (2020) Fluorinated α-helical polypeptides synchronize mucus permeation and cell penetration toward highly efficient pulmonary siRNA delivery against acute lung injury. Nano Letters 20, 1738–1746.CrossRefGoogle ScholarPubMed
Asadi, S et al. (2020) Efficacy of masks and face coverings in controlling outward aerosol particle emission from expiratory activities. Scientific Reports 10, 1–13.CrossRefGoogle ScholarPubMed
Kuijper, EC et al. (2021) Opportunities and challenges for antisense oligonucleotide therapies. Journal of Inherited Metabolic Disease 44, 72–87.CrossRefGoogle ScholarPubMed
Geary, RS (2009) Antisense oligonucleotide pharmacokinetics and metabolism. Expert Opinion on Drug Metabolism & Toxicology 5, 381–391.CrossRefGoogle ScholarPubMed