journal article·2024

Advances in carrier-delivered small interfering RNA based therapeutics for treatment of neurodegenerative diseases

Wendanqi Liang, Yi Luo, Ajing Xu, Jianjian Chu, Wenbo Ji, Li Wang, Yuankai Gu, Xinyu Lu, Along Hou, Yan Liu, Jie Gao, You Yin

Biomaterials Science· Volume 12· Issue 19
Publisher page

Abstract

Neurodegenerative diseases are devastating diseases that severely affect the health of people all over the world. RNA therapies have become one of the most promising critical drug treatments for neurodegenerative diseases due to their excellent gene and protein editing effects. However, the successful transport of RNA via the systemic route to the central nervous system remains one of the major obstacles in treating neurodegenerative diseases. This review will focus on therapeutic RNA that can successfully overcome the blood-brain barrier (BBB), with particular attention to small interfering RNAs (siRNAs), focusing on different types of neurodegenerative disease treatment strategies and accelerating their translation into clinical practice.
Source: europepmc · Quality: candidate unverified · Source record ↗

Research context

OpenAlex topics: RNA Interference and Gene Delivery · MicroRNA in disease regulation · Advanced biosensing and bioanalysis techniques

SciVX discovery tags are terminology-based. OpenAlex topics are source-provided classifications.

Affiliations

Department of Neurology, Second Affiliated Hospital (Shanghai Changzheng Hospital) of Naval Medical University, Shanghai, China

School of Health Science and Engineering, University of Shanghai for Science and Technology, Shanghai, China

New Drug Discovery and Development, Biotheus Inc., Zhuhai, China

Clinical Pharmacy Innovation Institute, Shanghai Jiao Tong University of Medicine, Shanghai, China

Department of Clinical Pharmacy, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China

Changhai Clinical Research Unit, Shanghai Changhai Hospital, Naval Medical University, Shanghai, China

Department of Neurology, Shanghai East Hospital, School of Medicine, Tongji University, Shanghai, China

Funding

Natural Science Foundation of Shanghai · Science and Technology Commission of Shanghai Municipality · National Natural Science Foundation of China

References (100+)

Show source-provided references
1. Feigin · Lancet Neurol. · 2019 · 18 · 45910.1016/S1474-4422(18)30499-X ↗
2. Batista · J. Neurol. Neurosci. · 2016 · 7 · 7410.21767/2171-6625.100074 ↗
3. Gitler · Dis. Models Mech. · 2017 · 10 · 49910.1242/dmm.030205 ↗
4. Alshaer · Eur. J. Pharmacol. · 2021 · 905 · 17417810.1016/j.ejphar.2021.174178 ↗
5. Cano · J. Nanobiotechnol. · 2021 · 19 · 12210.1186/s12951-021-00864-x ↗
6. Tuszynski · Alzheimer Dis. Assoc. Disord. · 2007 · 21 · 17910.1097/WAD.0b013e318068d6d2 ↗
7. Vejux · Int. J. Mol. Sci. · 2021 · 22 · 1365710.3390/ijms222413657 ↗
8. Ji · Mini-Rev. Med. Chem. · 2021 · 21 · 306210.2174/1389557521666210805110920 ↗
9. O'Connor · Trends Mol. Med. · 2015 · 21 · 50410.1016/j.molmed.2015.06.001 ↗
10. Alves · Hum. Gene Ther. · 2016 · 27 · 10010.1089/hum.2016.017 ↗
11. Serrano-Pozo · Lancet Neurol. · 2021 · 20 · 6810.1016/S1474-4422(20)30412-9 ↗
12. Kennington · Nat. Rev. Drug Discovery · 2009 · 8 · 27510.1038/nrd2855 ↗
13. Fromholt · Alzheimer's Res. Ther. · 2016 · 8 · 3610.1186/s13195-016-0202-1 ↗
14. . PubMed entry. [cited 2023 May 17]. Available from: https://www.ncbi.nlm.nih.gov/pubmed/35696026
15. Bhardwaj · J. Genet. Eng. Biotechnol. · 2021 · 19 · 12510.1186/s43141-021-00225-z ↗
16. Barman · Neurol. Ther. · 2020 · 9 · 41910.1007/s40120-020-00218-z ↗
17. Duarte · Front. Neurosci. · 2020 · 14 · 57906210.3389/fnins.2020.579062 ↗
18. Saw · Sci. China: Life Sci. · 2020 · 63 · 48510.1007/s11427-018-9438-y ↗
19. Gaynor · Chem. Soc. Rev. · 2010 · 39 · 416910.1039/b920362c ↗
20. Watts · Drug Discovery Today · 2008 · 13 · 84210.1016/j.drudis.2008.05.007 ↗
21. Whitehead · Nat. Rev. Drug Discovery · 2009 · 8 · 12910.1038/nrd2742 ↗
22. Sweeney · Physiol. Rev. · 2019 · 99 · 2110.1152/physrev.00050.2017 ↗
23. Yang · Small Weinh. Bergstr. Ger. · 2022 · 18 · e220318210.1002/smll.202203182 ↗
24. Seo · Biomedicines · 2023 · 11 · 195210.3390/biomedicines11071952 ↗
25. Spencer · Neurobiol. Dis. · 2019 · 127 · 16310.1016/j.nbd.2019.03.001 ↗
26. McKenzie · Proc. Natl. Acad. Sci. U. S. A. · 2018 · 115 · E606510.1073/pnas.1722041115 ↗
27. Holm · RNA Biol. · 2022 · 19 · 59410.1080/15476286.2022.2066334 ↗
28. Zhen · Cancer Gene Ther. · 2020 · 27 · 51510.1038/s41417-019-0141-7 ↗
29. Aimo · Nat. Rev. Cardiol. · 2022 · 19 · 65510.1038/s41569-022-00683-z ↗
30. Tao · Nanomedicine · 2019 · 14 · 51110.2217/nnm-2018-0522 ↗
31. Shchaslyvyi · Pharmacy · 2023 · 16 · 1416
32. Luttrell · Sci. Signaling · 2018 · 11 · eaat765010.1126/scisignal.aat7650 ↗
33. Hu · Signal Transduction Targeted Ther. · 2020 · 5 · 10110.1038/s41392-020-0207-x ↗
34. Niu · Asian J. Pharm. Sci. · 2019 · 14 · 48010.1016/j.ajps.2018.09.005 ↗
35. Liebner · Acta Neuropathol. · 2018 · 135 · 31110.1007/s00401-018-1815-1 ↗
36. Lee · Curr. Opin. Biotechnol · 2020 · 66 · 7810.1016/j.copbio.2020.06.009 ↗
37. Scherrmann · Vasc. Pharmacol. · 2002 · 38 · 34910.1016/S1537-1891(02)00202-1 ↗
38. Kulkarni · Acc. Chem. Res. · 2019 · 52 · 243510.1021/acs.accounts.9b00368 ↗
39. El-Andaloussi · Nat. Protoc. · 2012 · 7 · 211210.1038/nprot.2012.131 ↗
40. Tai · J. Controlled Release · 2019 · 307 · 9810.1016/j.jconrel.2019.06.022 ↗
41. Gooding · Chem. Biol. Drug Des. · 2012 · 80 · 78710.1111/cbdd.12052 ↗
42. Zahir-Jouzdani · J. Drug Delivery Sci. Technol. · 2018 · 45 · 42810.1016/j.jddst.2018.04.001 ↗
43. Mendonça · Mol. Pharmaceutics · 2021 · 18 · 149110.1021/acs.molpharmaceut.0c01238 ↗
44. Kadry · Fluids Barriers CNS · 2020 · 17 · 6910.1186/s12987-020-00230-3 ↗
45. Mendonça · Mol. Pharm. · 2021 · 18 · 149110.1021/acs.molpharmaceut.0c01238 ↗
46. Bellettato · Ital. J. Pediatr. · 2018 · 44 · 13110.1186/s13052-018-0563-0 ↗
47. Terstappen · Nat. Rev. Drug Discovery · 2021 · 20 · 36210.1038/s41573-021-00139-y ↗
48. Wong · Int. J. Mol. Sci. · 2019 · 20 · 38110.3390/ijms20020381 ↗
49. Li · J. Drug Targeting · 2017 · 25 · 1710.1080/1061186X.2016.1184272 ↗
50. van den Broek · Biomater. Adv. · 2022 · 141 · 21312510.1016/j.bioadv.2022.213125 ↗
51. Sheridan · Nat. Biotechnol. · 2019 · 37 · 70210.1038/d41587-019-00014-7 ↗
52. Orlacchio · Mini-Rev. Med. Chem. · 2007 · 7 · 116610.2174/138955707782331678 ↗
53. Lukashev · Biochemistry · 2016 · 81 · 700
54. Watanabe · Viruses · 2021 · 13 · 250210.3390/v13122502 ↗
55. Liu · Metab. Brain Dis. · 2021 · 36 · 4510.1007/s11011-020-00630-2 ↗
56. Maes · Neurosci. Lett. · 2019 · 707 · 13431010.1016/j.neulet.2019.134310 ↗
57. Zu · AAPS J. · 2021 · 23 · 7810.1208/s12248-021-00608-7 ↗
58. Thomas · Lancet Neurol. · 2005 · 4 · 60210.1016/S1474-4422(05)70188-5 ↗
59. Hu · Signal Transduction Targeted Ther. · 2020 · 5 · 10110.1038/s41392-020-0207-x ↗
60. Braasch · Bioorg. Med. Chem. Lett. · 2004 · 14 · 113910.1016/j.bmcl.2003.12.074 ↗
61. Detzer · Curr. Top. Med. Chem. · 2009 · 9 · 110910.2174/156802609789630884 ↗
62. Jahns · Nucleic Acids Res. · 2022 · 50 · 122110.1093/nar/gkab544 ↗
63. Selvam · Chem. Biol. Drug Des. · 2017 · 90 · 66510.1111/cbdd.12993 ↗
64. Shadid · Expert Opin. Drug Metab. Toxicol. · 2021 · 17 · 128110.1080/17425255.2021.1992382 ↗
65. Dong · Adv. Drug Delivery Rev. · 2019 · 144 · 13310.1016/j.addr.2019.05.004 ↗
66. Juliano · Nucleic Acids Res. · 2016 · 44 · 651810.1093/nar/gkw236 ↗
67. Chi · Drug Discovery Today · 2017 · 22 · 82310.1016/j.drudis.2017.01.013 ↗
68. Winkle · Nat. Rev. Drug Discovery · 2021 · 20 · 62910.1038/s41573-021-00219-z ↗
69. . PubMed entry. [cited 2024 Jul 1]. Available from: https://www.ncbi.nlm.nih.gov/pubmed/27939059
70. . PubMed entry. [cited 2024 Jul 1]. Available from: https://www.ncbi.nlm.nih.gov/pubmed/23907995
71. Schroeder · J. Intern. Med. · 2010 · 267 · 910.1111/j.1365-2796.2009.02189.x ↗
72. Cortes · Curr. Drug Targets · 2017 · 18 · 186610.2174/1389450118666170321130003 ↗
73. Moss · Mol. Pharm. · 2019 · 16 · 226510.1021/acs.molpharmaceut.8b01290 ↗
74. Buck · ACS Nano · 2019 · 13 · 375410.1021/acsnano.8b07858 ↗
75. Algarni · Biomater. Sci. · 2022 · 10 · 294010.1039/D2BM00168C ↗
76. Kulkarni · Acc. Chem. Res. · 2019 · 52 · 243510.1021/acs.accounts.9b00368 ↗
77. Sato · J. Controlled Release · 2019 · 295 · 14010.1016/j.jconrel.2019.01.001 ↗
78. Chen · Drug Delivery · 2016 · 23 · 136910.3109/10717544.2015.1038857 ↗
79. Khare · AAPS J. · 2021 · 24 · 810.1208/s12248-021-00653-2 ↗
80. Ashizawa · Cerebellum · 2019 · 18 · 9910.1007/s12311-018-0961-2 ↗
81. Alzheimer's Disease Neuroimaging Initiative · Alzheimer's Res. Ther. · 2021 · 13 · 1510.1186/s13195-020-00755-7 ↗
82. Rassu · Colloids Surf., B · 2017 · 152 · 29610.1016/j.colsurfb.2017.01.031 ↗
83. Tagalakis · Biomaterials · 2014 · 35 · 840610.1016/j.biomaterials.2014.06.003 ↗
84. Eygeris · Acc. Chem. Res. · 2022 · 55 · 210.1021/acs.accounts.1c00544 ↗
85. Hald Albertsen · Adv. Drug Delivery Rev. · 2022 · 188 · 11441610.1016/j.addr.2022.114416 ↗
86. Hirunagi · Mol. Ther.–Nucleic Acids · 2021 · 24 · 110.1016/j.omtn.2021.02.007 ↗
87. Rai · Polymers · 2019 · 11 · 74510.3390/polym11040745 ↗
88. Wang · Bioconjugate Chem. · 2019 · 30 · 32510.1021/acs.bioconjchem.8b00749 ↗
89. Xiang · Drug Discovery Today · 2017 · 22 · 131810.1016/j.drudis.2017.04.001 ↗
90. Chen · Bioconjugate Chem. · 2019 · 30 · 33810.1021/acs.bioconjchem.8b00688 ↗
91. Bitoque · Mater. Sci. Eng., C · 2018 · 93 · 27710.1016/j.msec.2018.07.071 ↗
92. Guizze · J. Pharm. Sci. · 2022 · 111 · 266210.1016/j.xphs.2022.07.009 ↗
93. Chu · Drug Delivery · 2022 · 29 · 210010.1080/10717544.2022.2094501 ↗
94. Bishop · J. Controlled Release · 2015 · 219 · 48810.1016/j.jconrel.2015.09.046 ↗
95. Zhou · Sci. Adv. · 2020 · 6 · eabc703110.1126/sciadv.abc7031 ↗
96. Gu · J. Controlled Release · 2020 · 322 · 3110.1016/j.jconrel.2020.03.009 ↗
97. Wang · J. Controlled Release · 2018 · 279 · 22010.1016/j.jconrel.2018.04.034 ↗
98. Gao · J. Nanobiotechnol. · 2017 · 15 · 3810.1186/s12951-017-0272-7 ↗
99. Jambhekar · Drug Discovery Today · 2016 · 21 · 35610.1016/j.drudis.2015.11.017 ↗
100. Jambhekar · Drug Discovery Today · 2016 · 21 · 36310.1016/j.drudis.2015.11.016 ↗