Login Register Cart 0
Generic placeholder image

Mini-Reviews in Medicinal Chemistry

Editor-in-Chief

ISSN (Print): 1389-5575
ISSN (Online): 1875-5607

Back
Journal Home About Journal Editorial Board Journal Insight Current Issue Volumes/Issues
Subscribe
Review Article

Nanoparticle-based Gene Therapy for Neurodegenerative Disorders

Author(s): Nelofer Ereej, Huma Hameed*, Mahtab Ahmad Khan, Saleha Faheem and Anam Hameed

Volume 24, Issue 19, 2024

Published on: 25 April, 2024

Page: [1723 - 1745] Pages: 23

DOI: 10.2174/0113895575301011240407082559

Price: $65

conference banner
Abstract

Neurological disorders present a formidable challenge in modern medicine due to the intricate obstacles set for the brain and the multipart nature of genetic interventions. This review article delves into the promising realm of nanoparticle-based gene therapy as an innovative approach to addressing the intricacies of neurological disorders. Nanoparticles (NPs) provide a multipurpose podium for the conveyance of therapeutic genes, offering unique properties such as precise targeting, enhanced stability, and the potential to bypass blood-brain barrier (BBB) restrictions. This comprehensive exploration reviews the current state of nanoparticle-mediated gene therapy in neurological disorders, highlighting recent advancements and breakthroughs. The discussion encompasses the synthesis of nanoparticles from various materials and their conjugation to therapeutic genes, emphasizing the flexibility in design that contributes to specific tissue targeting. The abstract also addresses the low immunogenicity of these nanoparticles and their stability in circulation, critical factors for successful gene delivery. While the potential of NP-based gene therapy for neurological disorders is vast, challenges and gaps in knowledge persist. The lack of extensive clinical trials leaves questions about safety and potential side effects unanswered. Therefore, this abstract emphasizes the need for further research to validate the therapeutic applications of NP-mediated gene therapy and to address nanosafety concerns. In conclusion, nanoparticle-based gene therapy emerges as a promising avenue in the pursuit of effective treatments for neurological disorders. This abstract advocates for continued research efforts to bridge existing knowledge gaps, unlocking the full potential of this innovative approach and paving the way for transformative solutions in the realm of neurological health.

Keywords: Nanoparticle, gene therapy, nanomedicine, neurological disorders, targeted, nanoparticles, blood-brain barrier.

« Previous Next »
Graphical Abstract

Organization W.H.; Neurological disorders: public health challenges 2006 Abed M.; A comprehensive examination of human brain disorders. J Biomed Sustain Healthcare Applic 2023,03(02),141-152 10.53759/0088/JBSHA202303014 Erkkinen M.G.; Kim M.O.; Geschwind M.D.; Clinical neurology and epidemiology of the major neurodegenerative diseases. Cold Spring Harb Perspect Biol 2018,10(4),a033118 10.1101/cshperspect.a033118 28716886 Genovese A.; Butler M.G.; Clinical assessment, genetics, and treatment approaches in autism spectrum disorder (ASD). Int J Mol Sci 2020,21(13),4726 10.3390/ijms21134726 32630718 Scheltens P.; Blennow K.; Breteler M.M.B.; de Strooper B.; Frisoni G.B.; Salloway S.; Van der Flier W.M.; Alzheimer’s disease. Lancet 2016,388(10043),505-517 10.1016/S0140-6736(15)01124-1 26921134 Teleanu D.M.; Niculescu A.G.; Lungu I.I.; Radu C.I.; Vladâcenco O.; Roza E.; Costăchescu B.; Grumezescu A.M.; Teleanu R.I.; An overview of oxidative stress, neuroinflammation, and neurodegenerative diseases. Int J Mol Sci 2022,23(11),5938 10.3390/ijms23115938 35682615 Esiaka D.; A mini-review of strategies for recruiting older African Americans to Alzheimer’s disease research. Community Health Equity Research and Policy 2022,0272684X221118493 Gutiérrez Á.; Cronin C.E.; Franz B.; Muniz-Terrera G.; Alzheimer’s Centers in US Hospitals: Enough to adequately address dementia care nationwide? J Aging Health 2023,08982643231200691 10.1177/08982643231200691 37699204 van Vliet E.F.; Knol M.J.; Schiffelers R.M.; Caiazzo M.; Fens M.H.A.M.; Levodopa-loaded nanoparticles for the treatment of Parkinson’s disease. J Control Release 2023,360,212-224 10.1016/j.jconrel.2023.06.026 37343725 Walter K.; What is acute ischemic stroke? JAMA 2022,327(9),885-885 10.1001/jama.2022.1420 35230392 Phipps M.S.; Cronin C.A.; Management of acute ischemic stroke. BMJ 2020,368,l6983 10.1136/bmj.l6983 32054610 Engel J.; Pedley T.A.; Introduction: what is epilepsyEpilepsy: a comprehensive textbook 2008,1-13 Chindo B.A.; Adzu B.; Gamaniel K.S.; Antiepileptic drug targets: An update on ion channels 2016 LaPenna P.; Tormoehlen L.M.; The pharmacology and toxicology of third-generation anticonvulsant drugs. J Med Toxicol 2017,13(4),329-342 10.1007/s13181-017-0626-4 28815428 Perucca E.; The new generation of antiepileptic drugs: Advantages and disadvantages. Br J Clin Pharmacol 1996,42(5),531-543 10.1111/j.1365-2125.1996.tb00046.x 8951184 Kohil A.; Jemmieh S.; Smatti M.K.; Yassine H.M.; Viral meningitis: an overview. Arch Virol 2021,166(2),335-345 10.1007/s00705-020-04891-1 33392820 Yekani M.; Memar M.Y.; Immunologic biomarkers for bacterial meningitis. Clin Chim Acta 2023,548,117470 10.1016/j.cca.2023.117470 37419301 Butowski N.A.; Epidemiology and diagnosis of brain tumors. Continuum (Minneap Minn), 21(2 Neuro-oncology) 2015,301-313 10.1212/01.CON.0000464171.50638.fa 25837897 Liu C.; Zong H.; Developmental origins of brain tumors. Curr Opin Neurobiol 2012,22(5),844-849 10.1016/j.conb.2012.04.012 22560511 Collins V.P.; Brain tumours: Classification and genes. J Neurol Neurosurg Psychiatry 2004,75(Suppl. 2),ii2-ii11 15146033 Tsung T.H.; Tsai Y.C.; Lee H.P.; Chen Y.H.; Lu D.W.; Biodegradable polymer-based drug-delivery systems for ocular diseases. Int J Mol Sci 2023,24(16),12976 10.3390/ijms241612976 37629157 Masserini M.; Nanoparticles for brain drug delivery. ISRN Biochem 2013,2013,238428 10.1155/2013/238428 Kaushik A.; Jayant R.D.; Sagar V.; Nair M.; The potential of magneto-electric nanocarriers for drug delivery. Expert Opin Drug Deliv 2014,11(10),1635-1646 10.1517/17425247.2014.933803 24986772 Vashist A.; Hydrogels: Stimuli responsive to on-demand drug delivery systems. Advances in Personalized Nanotherapeutics 2017,117-130 10.1007/978-3-319-63633-7_8 Colović M.B.; Krstić D.Z.; Lazarević-Pašti T.D.; Bondžić A.M.; Vasić V.M.; Acetylcholinesterase inhibitors: Pharmacology and toxicology. Curr Neuropharmacol 2013,11(3),315-335 10.2174/1570159X11311030006 24179466 McGleenon B.M.; Dynan K.B.; Passmore A.P.; Acetylcholinesterase inhibitors in Alzheimer’s disease. Br J Clin Pharmacol 1999,48(4),471-480 10.1046/j.1365-2125.1999.00026.x 10583015 Martín-Rapun R.; De Matteis L.; Ambrosone A.; Garcia-Embid S.; Gutierrez L.; de la Fuente J.M.; Targeted nanoparticles for the treatment of Alzheimer’s disease. Curr Pharm Des 2017,23(13),1927-1952 10.2174/1381612822666161226151011 28025949 Kuriakose D.; Xiao Z.; Pathophysiology and treatment of stroke: present status and future perspectives. Int J Mol Sci 2020,21(20),7609 10.3390/ijms21207609 33076218 Brott T.; Bogousslavsky J.; Treatment of acute ischemic stroke. N Engl J Med 2000,343(10),710-722 10.1056/NEJM200009073431007 10974136 Abou-Chebl A.; DeGeorgia M.A.; Andrefsky J.C.; Krieger D.W.; Technical refinements and drawbacks of a surface cooling technique for the treatment of severe acute ischemic stroke. Neurocrit Care 2004,1(2),131-144 10.1385/NCC:1:2:131 16174909 He W.; Zhang Z.; Sha X.; Nanoparticles-mediated emerging approaches for effective treatment of ischemic stroke. Biomaterials 2021,277,121111 10.1016/j.biomaterials.2021.121111 34488117 Cai R.; Chen C.; The crown and the scepter: Roles of the protein corona in nanomedicine. Adv Mater 2019,31(45),1805740 10.1002/adma.201805740 30589115 Nadkarni S.; LaJoie J.; Devinsky O.; Current treatments of epilepsy. Neurology 2005,64(12)(Suppl. 3),S2-S11 15994220 Cascino G.D.; Surgical treatment for epilepsy. Epilepsy Res 2004,60(2-3),179-186 10.1016/j.eplepsyres.2004.07.003 15380562 Löscher W.; Potschka H.; Sisodiya S.M.; Vezzani A.; Drug resistance in epilepsy: Clinical impact, potential mechanisms, and new innovative treatment options. Pharmacol Rev 2020,72(3),606-638 10.1124/pr.120.019539 32540959 Bonilla L.; Esteruelas G.; Ettcheto M.; Espina M.; García M.L.; Camins A.; Souto E.B.; Cano A.; Sánchez-López E.; Biodegradable nanoparticles for the treatment of epilepsy: From current advances to future challenges. Epilepsia Open 2022,7(Suppl. 1),S121-S132 10.1002/epi4.12567 34862851 Balestrino R.; Schapira A.H.V.; Parkinson disease. Eur J Neurol 2020,27(1),27-42 10.1111/ene.14108 31631455 Beitz J.M.; Parkinson s disease a review. Front Biosci (Schol Ed) 2014,S6(1),65-74 10.2741/S415 24389262 Baskin J.; Jeon J.E.; Lewis S.J.G.; Nanoparticles for drug delivery in Parkinson’s disease. J Neurol 2021,268(5),1981-1994 10.1007/s00415-020-10291-x 33141248 Paleacu D.; Tetrabenazine in the treatment of Huntington’s disease. Neuropsychiatr Dis Treat 2007,3(5),545-551 19381278 Frank S.; Treatment of Huntington’s disease. Neurotherapeutics 2014,11(1),153-160 10.1007/s13311-013-0244-z 24366610 Bonelli R.M.; Wenning G.K.; Kapfhammer H.P.; Huntington’s disease: Present treatments and future therapeutic modalities. Int Clin Psychopharmacol 2004,19(2),51-62 10.1097/00004850-200403000-00001 15076012 Singh S.; Hema; Sharma, N.; Sachdeva, M.; Behl, T.; Zahoor, I.; Fuloria, N.K.; Sekar, M.; Fuloria, S.; Subramaniyan, V.; Alsubayiel, A.M.; Dailah, H.G.; Naved, T.; Bhatia, S.; Al-Harrasi, A.; Aleya, L. Focusing the pivotal role of nanotechnology in Huntington’s disease: An insight into the recent advancements. Environ Sci Pollut Res Int 2022,29(49),73809-73827 10.1007/s11356-022-22830-2 36100788 Hauser S.L.; Cree B.A.; Treatment of multiple sclerosis: A review. Am J Med 2020,133(12),1380-1390.e2 10.1016/j.amjmed.2020.05.049 Rafiee Zadeh A.; Ghadimi K.; Ataei A.; Askari M.; Sheikhinia N.; Tavoosi N.; Falahatian M.; Mechanism and adverse effects of multiple sclerosis drugs: A review article. Part 2. Int J Physiol Pathophysiol Pharmacol 2019,11(4),105-114 31523358 Ballerini C.; Baldi G.; Aldinucci A.; Maggi P.; Nanomaterial applications in multiple sclerosis inflamed brain. J Neuroimmune Pharmacol 2015,10(1),1-13 10.1007/s11481-015-9588-y 25616566 Owonikoko T.K.; Arbiser J.; Zelnak A.; Shu H.K.G.; Shim H.; Robin A.M.; Kalkanis S.N.; Whitsett T.G.; Salhia B.; Tran N.L.; Ryken T.; Moore M.K.; Egan K.M.; Olson J.J.; Current approaches to the treatment of metastatic brain tumours. Nat Rev Clin Oncol 2014,11(4),203-222 10.1038/nrclinonc.2014.25 24569448 Amjad M.T.; Chidharla A.; Kasi A.; Gan To Kagaku Ryoho 2020 Kang C.; Sun Y.; Zhu J.; Li W.; Zhang A.; Kuang T.; Xie J.; Yang Z.; Delivery of nanoparticles for treatment of brain tumor. Curr Drug Metab 2016,17(8),745-754 10.2174/1389200217666160728152939 27469219 Chaudhuri T.R.; Straubinger R.M.; Nanoparticles for brain tumor delivery Nervous system drug delivery 2019,229-250 10.1016/B978-0-12-813997-4.00012-8 Carson M.J.; Cameron Thrash J.; Walter B.; The cellular response in neuroinflammation: The role of leukocytes, microglia and astrocytes in neuronal death and survival. Clin Neurosci Res 2006,6(5),237-245 10.1016/j.cnr.2006.09.004 19169437 Harder B.G.; Blomquist M.R.; Wang J.; Kim A.J.; Woodworth G.F.; Winkles J.A.; Loftus J.C.; Tran N.L.; Developments in blood-brain barrier penetrance and drug repurposing for improved treatment of glioblastoma. Front Oncol 2018,8,462 10.3389/fonc.2018.00462 30406029 Abbott N.J.; Astrocyte–endothelial interactions and blood–brain barrier permeability. J Anat 2002,200(6),629-638 10.1046/j.1469-7580.2002.00064.x 12162730 Do Thi N.A.; Saillour P.; Ferrero L.; Dedieu J.F.; Mallet J.; Paunio T.; Delivery of GDNF by an E1,E3/E4 deleted adenoviral vector and driven by a GFAP promoter prevents dopaminergic neuron degeneration in a rat model of Parkinson’s disease. Gene Ther 2004,11(9),746-756 10.1038/sj.gt.3302222 14724667 Hocquemiller M.; Giersch L.; Audrain M.; Parker S.; Cartier N.; Adeno-associated virus-based gene therapy for CNS diseases. Hum Gene Ther 2016,27(7),478-496 10.1089/hum.2016.087 27267688 Ganipineni L.P.; Danhier F.; Préat V.; Drug delivery challenges and future of chemotherapeutic nanomedicine for glioblastoma treatment. J Control Release 2018,281,42-57 10.1016/j.jconrel.2018.05.008 29753958 Scarpa M.; Orchard P.J.; Schulz A.; Dickson P.I.; Haskins M.E.; Escolar M.L.; Giugliani R.; Treatment of brain disease in the mucopolysaccharidoses. Mol Genet Metab 2017,122,25-34 10.1016/j.ymgme.2017.10.007 29153844 Pardridge W.M.; Drug and gene delivery to the brain: The vascular route. Neuron 2002,36(4),555-558 10.1016/S0896-6273(02)01054-1 12441045 van Tellingen O.; Yetkin-Arik B.; de Gooijer M.C.; Wesseling P.; Wurdinger T.; de Vries H.E.; Overcoming the blood–brain tumor barrier for effective glioblastoma treatment. Drug Resist Updat 2015,19,1-12 10.1016/j.drup.2015.02.002 25791797 Jones C.H.; Chen C.K.; Ravikrishnan A.; Rane S.; Pfeifer B.A.; Overcoming nonviral gene delivery barriers: Perspective and future. Mol Pharm 2013,10(11),4082-4098 10.1021/mp400467x 24093932 Joshi C.R.; Labhasetwar V.; Ghorpade A.; Destination brain: The past, present, and future of therapeutic gene delivery. J Neuroimmune Pharmacol 2017,12(1),51-83 10.1007/s11481-016-9724-3 28160121 Mali S.; Delivery systems for gene therapy. Indian J Hum Genet 2013,19(1),3-8 10.4103/0971-6866.112870 23901186 Kantor B.; McCown T.; Leone P.; Gray S.J.; Clinical applications involving CNS gene transfer. Adv Genet 2014,87,71-124 10.1016/B978-0-12-800149-3.00002-0 25311921 Gray S.J.; Matagne V.; Bachaboina L.; Yadav S.; Ojeda S.R.; Samulski R.J.; Preclinical differences of intravascular AAV9 delivery to neurons and glia: A comparative study of adult mice and nonhuman primates. Mol Ther 2011,19(6),1058-1069 10.1038/mt.2011.72 21487395 Ma C.C.; Wang Z.L.; Xu T.; He Z.Y.; Wei Y.Q.; The approved gene therapy drugs worldwide: from 1998 to 2019. Biotechnol Adv 2020,40,107502 10.1016/j.biotechadv.2019.107502 31887345 Bulaklak K.; Gersbach C.A.; The once and future gene therapy. Nat Commun 2020,11(1),5820 10.1038/s41467-020-19505-2 33199717 Wirth T.; Parker N.; Ylä-Herttuala S.; History of gene therapy. Gene 2013,525(2),162-169 10.1016/j.gene.2013.03.137 23618815 Bayle J.H.; Grimley J.S.; Stankunas K.; Gestwicki J.E.; Wandless T.J.; Crabtree G.R.; Rapamycin analogs with differential binding specificity permit orthogonal control of protein activity. Chem Biol 2006,13(1),99-107 10.1016/j.chembiol.2005.10.017 16426976 Zu H.; Gao D.; Non-viral vectors in gene therapy: Recent development, challenges, and prospects. AAPS J 2021,23(4),78 10.1208/s12248-021-00608-7 34076797 Manfredsson F.P.; Mandel R.J.; Development of gene therapy for neurological disorders. Discov Med 2010,9(46),204-211 20350486 Iqubal A.; Iqubal M.K.; Khan A.; Ali J.; Baboota S.; Haque S.E.; Gene therapy, a novel therapeutic tool for neurological disorders: Current progress, challenges and future prospective. Curr Gene Ther 2020,20(3),184-194 10.2174/1566523220999200716111502 32674730 Simonato M.; Bennett J.; Boulis N.M.; Castro M.G.; Fink D.J.; Goins W.F.; Gray S.J.; Lowenstein P.R.; Vandenberghe L.H.; Wilson T.J.; Wolfe J.H.; Glorioso J.C.; Progress in gene therapy for neurological disorders. Nat Rev Neurol 2013,9(5),277-291 10.1038/nrneurol.2013.56 23609618 Shirley J.L.; Herzog R.W.; AAV immunogenicity: New answers create new questions. Mol Ther 2018,26(11),2538-2539 10.1016/j.ymthe.2018.10.004 30366819 Marrone L.; Marchi P.M.; Azzouz M.; Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders. Expert Opin Biol Ther 2022,22(9),1163-1176 10.1080/14712598.2022.2012148 34904932 Neuwelt E.A.; Pagel M.A.; Geller A.; Muldoon L.L.; Gene replacement therapy in the central nervous system: Viral vector-mediated therapy of global neurodegenerative disease. Behav Brain Sci 1995,18(1),1-9 10.1017/S0140525X00037237 Boonanuntanasarn S.; Gene knockdown: A powerful tool for gene function study in fish. J World Aquacult Soc 2008,39(3),311-323 10.1111/j.1749-7345.2008.00167.x Mandel R.J.; Manfredsson F.P.; Foust K.D.; Rising A.; Reimsnider S.; Nash K.; Burger C.; Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders. Mol Ther 2006,13(3),463-483 10.1016/j.ymthe.2005.11.009 16412695 Meng Y.; Pople C.B.; Lea-Banks H.; Abrahao A.; Davidson B.; Suppiah S.; Vecchio L.M.; Samuel N.; Mahmud F.; Hynynen K.; Hamani C.; Lipsman N.; Safety and efficacy of focused ultrasound induced blood-brain barrier opening, an integrative review of animal and human studies. J Control Release 2019,309,25-36 10.1016/j.jconrel.2019.07.023 31326464 Marks W.J.; Ostrem J.L.; Verhagen L.; Starr P.A.; Larson P.S.; Bakay R.A.E.; Taylor R.; Cahn-Weiner D.A.; Stoessl A.J.; Olanow C.W.; Bartus R.T.; Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2–neurturin) to patients with idiopathic Parkinson’s disease: An open-label, phase I trial. Lancet Neurol 2008,7(5),400-408 10.1016/S1474-4422(08)70065-6 18387850 Jenkins E.P.W.; Finch A.; Gerigk M.; Triantis I.F.; Watts C.; Malliaras G.G.; Electrotherapies for glioblastoma. Adv Sci (Weinh) 2021,8(18),2100978 10.1002/advs.202100978 34292672 Khaddour K.; Johanns T.; Ansstas G.; The landscape of novel therapeutics and challenges in glioblastoma multiforme: Contemporary state and future directions. Pharmaceuticals (Basel) 2020,13(11),389 10.3390/ph13110389 33202642 Gilligan K.C.; Protecting consumers and regulating data: The need for comprehensive federal oversight of the direct-to-consumer genetic testing industry. Drexel L Rev 2022,14,207 Thomas C.E.; Ehrhardt A.; Kay M.A.; Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet 2003,4(5),346-358 10.1038/nrg1066 12728277 Srejovic N.; Copyright protection for works in the language of life. Wash Law Rev 2022,97,459 Al-Dosari M.S.; Gao X.; Nonviral gene delivery: Principle, limitations, and recent progress. AAPS J 2009,11(4),671-681 10.1208/s12248-009-9143-y 19834816 Tortajada L.; Felip-León C.; Vicent M.J.; Polymer-based non-viral vectors for gene therapy in the skin. Polym Chem 2022,13(6),718-735 10.1039/D1PY01485D Zhu H.; Luo H.; Chang R.; Yang Y.; Liu D.; Ji Y.; Qin H.; Rong H.; Yin J.; Protein-based delivery systems for RNA delivery. J Control Release 2023,363,253-274 10.1016/j.jconrel.2023.09.032 37741460 Ibraheem D.; Elaissari A.; Fessi H.; Gene therapy and DNA delivery systems. Int J Pharm 2014,459(1-2),70-83 10.1016/j.ijpharm.2013.11.041 24286924 Choudhury S.R.; Hudry E.; Maguire C.A.; Sena-Esteves M.; Breakefield X.O.; Grandi P.; Viral vectors for therapy of neurologic diseases. Neuropharmacology 2017,120,63-80 10.1016/j.neuropharm.2016.02.013 26905292 Chira S.; Jackson C.S.; Oprea I.; Ozturk F.; Pepper M.S.; Diaconu I.; Braicu C.; Raduly L.Z.; Calin G.A.; Berindan-Neagoe I.; Progresses towards safe and efficient gene therapy vectors. Oncotarget 2015,6(31),30675-30703 10.18632/oncotarget.5169 26362400 Guo X.; Huang L.; Recent advances in nonviral vectors for gene delivery. Acc Chem Res 2012,45(7),971-979 10.1021/ar200151m 21870813 Jayant R.D.; Sosa D.; Kaushik A.; Atluri V.; Vashist A.; Tomitaka A.; Nair M.; Current status of non-viral gene therapy for CNS disorders. Expert Opin Drug Deliv 2016,13(10),1433-1445 10.1080/17425247.2016.1188802 27249310 Maguire C.A.; Ramirez S.H.; Merkel S.F.; Sena-Esteves M.; Breakefield X.O.; Gene therapy for the nervous system: Challenges and new strategies. Neurotherapeutics 2014,11(4),817-839 10.1007/s13311-014-0299-5 25159276 Zhao Y.; Huang L.; Lipid nanoparticles for gene delivery. Adv Genet 2014,88,13-36 10.1016/B978-0-12-800148-6.00002-X 25409602 Bergen J.M.; Park I.K.; Horner P.J.; Pun S.H.; Nonviral approaches for neuronal delivery of nucleic acids. Pharm Res 2008,25(5),983-998 10.1007/s11095-007-9439-5 17932730 Yin J.; Wang Q.; Hou S.; Bao L.; Yao W.; Gao X.; Potent protein delivery into mammalian cells via a supercharged polypeptide. J Am Chem Soc 2018,140(49),17234-17240 10.1021/jacs.8b10299 30398334 Gray D.; Gene Editing of Bruton’s Tyrosine Kinase for Treatment of X-Linked Agammaglobulinemia 2020 Pierce E.A.; Bennett J.; The status of RPE65 gene therapy trials: Safety and efficacy. Cold Spring Harb Perspect Med 2015,5(9),a017285 10.1101/cshperspect.a017285 25635059 Healy S.; Bakuzis A.F.; Goodwill P.W.; Attaluri A.; Bulte J.W.M.; Ivkov R.; Clinical magnetic hyperthermia requires integrated magnetic particle imaging. Wiley Interdiscip Rev Nanomed Nanobiotechnol 2022,14(3),e1779 10.1002/wnan.1779 35238181 OLI Nano based approach for the neurological disorder treatments-a scenario (A-Review). Orient J Chem 2023,39(2) Bremer-Hoffmann S.; Amenta V.; Rossi F.; Nanomedicines in the European translational process. Eur J Nanomed 2015,7(3),191-202 10.1515/ejnm-2015-0027 Li H.; Feng Y.; Luo Q.; Li Z.; Li X.; Gan H.; Gu Z.; Gong Q.; Luo K.; Stimuli-activatable nanomedicine meets cancer theranostics. Theranostics 2023,13(15),5386-5417 10.7150/thno.87854 37908735 Lasher S.Y.; Synthesis, characterization and anticancer effects of Quantum dots in neuroblastoma and glioblastoma cell lines 2018 Buzea C.; Pacheco I.I.; Robbie K.; Nanomaterials and nanoparticles: Sources and toxicity. Biointerphases 2007,2(4),MR17-MR71 10.1116/1.2815690 20419892 Martínez-Ballesta M.C.; Gil-Izquierdo Á.; García-Viguera C.; Domínguez-Perles R.; Nanoparticles and controlled delivery for bioactive compounds: Outlining challenges for new “smart-foods” for health. Foods 2018,7(5),72 10.3390/foods7050072 29735897 Stimphil E.; Nagesetti A.; Guduru R.; Stewart T.; Rodzinski A.; Liang P.; Khizroev S.; Physics considerations in targeted anticancer drug delivery by magnetoelectric nanoparticles. Appl Phys Rev 2017,4(2),021101 10.1063/1.4978642 Vieira D.; Gamarra L.; Getting into the brain: Liposome-based strategies for effective drug delivery across the blood–brain barrier. Int J Nanomedicine 2016,11,5381-5414 10.2147/IJN.S117210 27799765 Laidmäe I.; Meos A.; Kjærvik I.A.; Ingebrigtsen S.G.; Škalko-Basnet N.; Kirsimäe K.; Romann T.; Joost U.; Kisand V.; Kogermann K.; Electrospun amphiphilic nanofibers as templates for in situ preparation of chloramphenicol-loaded liposomes. Pharmaceutics 2021,13(11),1742 10.3390/pharmaceutics13111742 34834157 Othman A.K.; El Kurdi R.; Badran A.; Mesmar J.; Baydoun E.; Patra D.; Liposome-based nanocapsules for the controlled release of dietary curcumin: PDDA and silica nanoparticle-coated DMPC liposomes enhance the fluorescence efficiency and anticancer activity of curcumin. RSC Advances 2022,12(18),11282-11292 10.1039/D2RA00071G 35425076 Abbott N.J.; Rönnbäck L.; Hansson E.; Astrocyte–endothelial interactions at the blood–brain barrier. Nat Rev Neurosci 2006,7(1),41-53 10.1038/nrn1824 16371949 Posadas I.; Monteagudo S.; Ceña V.; Nanoparticles for brain-specific drug and genetic material delivery, imaging and diagnosis. Nanomedicine (Lond) 2016,11(7),833-849 10.2217/nnm.16.15 26980585 Xu G.; Mahajan S.; Roy I.; Yong K.T.; Theranostic quantum dots for crossing blood–brain barrier in vitro and providing therapy of HIV-associated encephalopathy. Front Pharmacol 2013,4,140 10.3389/fphar.2013.00140 24298256 Shilo M.; Motiei M.; Hana P.; Popovtzer R.; Transport of nanoparticles through the blood–brain barrier for imaging and therapeutic applications. Nanoscale 2014,6(4),2146-2152 10.1039/C3NR04878K 24362586 Demeule M.; Currie J.C.; Bertrand Y.; Ché C.; Nguyen T.; Régina A.; Gabathuler R.; Castaigne J.P.; Béliveau R.; Involvement of the low‐density lipoprotein receptor‐related protein in the transcytosis of the brain delivery vector Angiopep‐2. J Neurochem 2008,106(4),1534-1544 10.1111/j.1471-4159.2008.05492.x 18489712 Nguyen T.T.; Dung Nguyen T.T.; Vo T.K.; Tran N.M.A.; Nguyen M.K.; Van Vo T.; Van Vo G.; Nanotechnology-based drug delivery for central nervous system disorders. Biomed Pharmacother 2021,143,112117 10.1016/j.biopha.2021.112117 34479020 Pires P.C.; Santos A.O.; Nanosystems in nose-to-brain drug delivery: A review of non-clinical brain targeting studies. J Control Release 2018,270,89-100 10.1016/j.jconrel.2017.11.047 29199063 Grassin-Delyle S.; Buenestado A.; Naline E.; Faisy C.; Blouquit-Laye S.; Couderc L.J.; Le Guen M.; Fischler M.; Devillier P.; Intranasal drug delivery: An efficient and non-invasive route for systemic administration. Pharmacol Ther 2012,134(3),366-379 10.1016/j.pharmthera.2012.03.003 22465159 Fan Y.; Updated progress of nanocarrier-based intranasal drug delivery systems for treatment of brain diseases. Crit Rev Ther Drug Carrier Syst 2018,35(5),433-467 10.1615/CritRevTherDrugCarrierSyst.2018024697 Samaridou E.; Alonso M.J.; Nose-to-brain peptide delivery – The potential of nanotechnology. Bioorg Med Chem 2018,26(10),2888-2905 10.1016/j.bmc.2017.11.001 29170026 Porfiryeva N.N.; Semina I.I.; Salakhov I.A.; Moustafine R.I.; Khutoryanskiy V.V.; Mucoadhesive and mucus-penetrating interpolyelectrolyte complexes for nose-to-brain drug delivery. Nanomedicine 2021,37,102432 10.1016/j.nano.2021.102432 34186258 Bruinsmann F.; Pigana S.; Aguirre T.; Souto G.; Pereira G.; Bianchera A.; Fasiolo L.; Colombo G.; Marques M.; Pohlmann A.; Guterres S.; Sonvico F.; Chitosan-coated nanoparticles: Effect of chitosan molecular weight on nasal transmucosal delivery. Pharmaceutics 2019,11(2),86 10.3390/pharmaceutics11020086 30781722 Shrestha N.; Khan S.; Neupane Y.R.; Dang S.; Md S.; Fahmy U.A.; Kotta S.; Alhakamy N.A.; Baboota S.; Ali J.; Tailoring midazolam-loaded chitosan nanoparticulate formulation for enhanced brain delivery via intranasal route. Polymers (Basel) 2020,12(11),2589 10.3390/polym12112589 33158148 Zada M.H.; Kubek M.; Khan W.; Kumar A.; Domb A.; Dispersible hydrolytically sensitive nanoparticles for nasal delivery of thyrotropin releasing hormone (TRH). J Control Release 2019,295,278-289 10.1016/j.jconrel.2018.12.050 30610951 Agrawal M.; Saraf S.; Saraf S.; Dubey S.K.; Puri A.; Gupta U.; Kesharwani P.; Ravichandiran V.; Kumar P.; Naidu V.G.M.; Murty U.S.; Ajazuddin; Alexander, A. Stimuli-responsive in situ gelling system for nose-to-brain drug delivery. J Control Release 2020,327,235-265 10.1016/j.jconrel.2020.07.044 32739524 Arumugam K.; Subramanian G.; Mallayasamy S.; Averineni R.; Reddy M.; Udupa N.; A study of rivastigmine liposomes for delivery into the brain through intranasal route. Acta Pharm 2008,58(3),287-297 10.2478/v10007-008-0014-3 19103565 Rompicherla S.K.L.; Arumugam K.; Bojja S.L.; Kumar N.; Rao C.M.; Pharmacokinetic and pharmacodynamic evaluation of nasal liposome and nanoparticle based rivastigmine formulations in acute and chronic models of Alzheimer’s disease. Naunyn Schmiedebergs Arch Pharmacol 2021,394(8),1737-1755 10.1007/s00210-021-02096-0 34086100 Puri A.; Lipid-based nanoparticles as pharmaceutical drug carriers: From concepts to clinic. Crit Rev Ther Drug Carrier Syst 2009,26(6),523-580 10.1615/CritRevTherDrugCarrierSyst.v26.i6.10 Anderluzzi G.; Lou G.; Su Y.; Perrie Y.; Scalable manufacturing processes for solid lipid nanoparticles. Pharm Nanotechnol 2019,7(6),444-459 10.2174/2211738507666190925112942 31840610 Gupta S.; Systematic approach for the formulation and optimization of solid lipid nanoparticles of efavirenz by high pressure homogenization using design of experiments for brain targeting and enhanced bioavailability. BioMed Res Int 2017,2017,5984014 10.1155/2017/5984014 Fu Q.; Li Z.; Fu F.; Chen X.; Song J.; Yang H.; Stimuli-responsive plasmonic assemblies and their biomedical applications. Nano Today 2021,36,101014 10.1016/j.nantod.2020.101014 33250931 Sukumar U.K.; Bose R.J.C.; Malhotra M.; Babikir H.A.; Afjei R.; Robinson E.; Zeng Y.; Chang E.; Habte F.; Sinclair R.; Gambhir S.S.; Massoud T.F.; Paulmurugan R.; Intranasal delivery of targeted polyfunctional gold–iron oxide nanoparticles loaded with therapeutic microRNAs for combined theranostic multimodality imaging and presensitization of glioblastoma to temozolomide. Biomaterials 2019,218,119342 10.1016/j.biomaterials.2019.119342 31326657 Arruebo M.; Fernández-Pacheco R.; Ibarra M.R.; Santamaría J.; Magnetic nanoparticles for drug delivery. Nano Today 2007,2(3),22-32 10.1016/S1748-0132(07)70084-1 Jafari S.; Mair L.O.; Weinberg I.N.; Baker-McKee J.; Hale O.; Watson-Daniels J.; English B.; Stepanov P.Y.; Ropp C.; Atoyebi O.F.; Sun D.; Magnetic drilling enhances intra-nasal transport of particles into rodent brain. J Magn Magn Mater 2019,469,302-305 10.1016/j.jmmm.2018.08.048 Vallet-Regí M.; Colilla M.; Izquierdo-Barba I.; Manzano M.; Mesoporous silica nanoparticles for drug delivery: Current insights. Molecules 2017,23(1),47 10.3390/molecules23010047 29295564 Lungare S.; Hallam K.; Badhan R.K.S.; Phytochemical-loaded mesoporous silica nanoparticles for nose-to-brain olfactory drug delivery. Int J Pharm 2016,513(1-2),280-293 10.1016/j.ijpharm.2016.09.042 27633279 Hoseini-Ghahfarokhi M.; Mirkiani S.; Mozaffari N.; Abdolahi Sadatlu M.A.; Ghasemi A.; Abbaspour S.; Akbarian M.; Farjadain F.; Karimi M.; Applications of graphene and graphene oxide in smart drug/gene delivery: Is the world still flat? Int J Nanomedicine 2020,15,9469-9496 10.2147/IJN.S265876 33281443 Newman L.; Rodrigues A.F.; Jasim D.A.; Vacchi I.A.; Ménard-Moyon C.; Bianco A.; Bussy C.; Kostarelos K.; Nose-to-brain translocation and cerebral biodegradation of thin graphene oxide nanosheets. Cell Rep Phys Sci 2020,1(9),100176 10.1016/j.xcrp.2020.100176 Hopkins L.E.; Patchin E.S.; Chiu P.L.; Brandenberger C.; Smiley-Jewell S.; Pinkerton K.E.; Nose-to-brain transport of aerosolised quantum dots following acute exposure. Nanotoxicology 2014,8(8),885-893 10.3109/17435390.2013.842267 24040866 Junghanns J-U.A.; Müller R.H.; Nanocrystal technology, drug delivery and clinical applications. Int J Nanomedicine 2008,3(3),295-309 18990939 Mauludin R.; Müller R.H.; Keck C.M.; Development of an oral rutin nanocrystal formulation. Int J Pharm 2009,370(1-2),202-209 10.1016/j.ijpharm.2008.11.029 19114097 Lu Y.; Qi J.; Dong X.; Zhao W.; Wu W.; The in vivo fate of nanocrystals. Drug Discov Today 2017,22(4),744-750 10.1016/j.drudis.2017.01.003 28088442 Zhao J.; Liu Y.; Wang L.; Zhou Y.; Du J.; Wang Y.; Functional and modified nanocrystals technology for target drug delivery. J Nanosci Nanotechnol 2018,18(8),5207-5221 10.1166/jnn.2018.15421 29458570 Slezak A.; Chang K.; Hossainy S.; Mansurov A.; Rowan S.J.; Hubbell J.A.; Guler M.O.; Therapeutic synthetic and natural materials for immunoengineering. Chem Soc Rev 2024,53(4),1789-1822 10.1039/D3CS00805C 38170619 Sim T.M.; Tarini D.; Dheen S.T.; Bay B.H.; Srinivasan D.K.; Nanoparticle-based technology approaches to the management of neurological disorders. Int J Mol Sci 2020,21(17),6070 10.3390/ijms21176070 32842530 Tachibana M.; Amato P.; Sparman M.; Woodward J.; Sanchis D.M.; Ma H.; Gutierrez N.M.; Tippner-Hedges R.; Kang E.; Lee H.S.; Ramsey C.; Masterson K.; Battaglia D.; Lee D.; Wu D.; Jensen J.; Patton P.; Gokhale S.; Stouffer R.; Mitalipov S.; Towards germline gene therapy of inherited mitochondrial diseases. Nature 2013,493(7434),627-631 10.1038/nature11647 23103867 Shen F.; Fan Y.; Su H.; Zhu Y.; Chen Y.; Liu W.; Young W.L.; Yang G-Y.; Adeno-associated viral vector-mediated hypoxia-regulated VEGF gene transfer promotes angiogenesis following focal cerebral ischemia in mice. Gene Ther 2008,15(1),30-39 10.1038/sj.gt.3303048 17960159 Thrasher A.J.; Williams D.A.; Evolving gene therapy in primary immunodeficiency. Mol Ther 2017,25(5),1132-1141 10.1016/j.ymthe.2017.03.018 28366768 Ross C.A.; Aylward E.H.; Wild E.J.; Langbehn D.R.; Long J.D.; Warner J.H.; Scahill R.I.; Leavitt B.R.; Stout J.C.; Paulsen J.S.; Reilmann R.; Unschuld P.G.; Wexler A.; Margolis R.L.; Tabrizi S.J.; Huntington disease: Natural history, biomarkers and prospects for therapeutics. Nat Rev Neurol 2014,10(4),204-216 10.1038/nrneurol.2014.24 24614516 Isik A.T.; Late onset Alzheimer’s disease in older people. Clin Interv Aging 2010,5,307-311 10.2147/CIA.S11718 21103401 Benatar M.; Wuu J.; Andersen P.M.; Atassi N.; David W.; Cudkowicz M.; Schoenfeld D.; Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive SOD1 ALS. Neurology 2018,90(7),e565-e574 10.1212/WNL.0000000000004960 29367439 Sinnett S.E.; Hector R.D.; Gadalla K.K.E.; Heindel C.; Chen D.; Zaric V.; Bailey M.E.S.; Cobb S.R.; Gray S.J.; Improved MECP2 gene therapy extends the survival of MeCP2-null mice without apparent toxicity after intracisternal delivery. Mol Ther Methods Clin Dev 2017,5,106-115 10.1016/j.omtm.2017.04.006 28497072 Herrera-Carrillo E.; Liu Y.P.; Berkhout B.; Improving miRNA delivery by optimizing miRNA expression cassettes in diverse virus vectors. Hum Gene Ther Methods 2017,28(4),177-190 10.1089/hgtb.2017.036 28712309 Yao W.; Guo G.; Zhang Q.; Fan L.; Wu N.; Bo Y.; The application of multiple miRNA response elements enables oncolytic adenoviruses to possess specificity to glioma cells. Virology 2014,458-459,69-82 10.1016/j.virol.2014.04.007 24928040 Bofill-De Ros X.; Rovira-Rigau M.; Fillat C.; Implications of MicroRNAs in oncolytic virotherapy. Front Oncol 2017,7,142 10.3389/fonc.2017.00142 28725635 Pérez-Martínez F.C.; Carrión B.; Ceña V.; The use of nanoparticles for gene therapy in the nervous system. J Alzheimers Dis 2012,31(4),697-710 10.3233/JAD-2012-120661 22695620 Zhao N.; Yang X.; Calvelli H.R.; Cao Y.; Francis N.L.; Chmielowski R.A.; Joseph L.B.; Pang Z.P.; Uhrich K.E.; Baum J.; Moghe P.V.; Antioxidant nanoparticles for concerted inhibition of α-synuclein fibrillization, and attenuation of microglial intracellular aggregation and activation. Front Bioeng Biotechnol 2020,8,112 10.3389/fbioe.2020.00112 32154238 Zhang G.; Xia Y.; Wan F.; Ma K.; Guo X.; Kou L.; Yin S.; Han C.; Liu L.; Huang J.; Xiong N.; Wang T.; New perspectives on roles of alpha-synuclein in Parkinson’s disease. Front Aging Neurosci 2018,10,370 10.3389/fnagi.2018.00370 30524265 Zhang W.; Wang W.; Yu D.X.; Xiao Z.; He Z.; Application of nanodiagnostics and nanotherapy to CNS diseases. Nanomedicine (Lond) 2018,13(18),2341-2371 10.2217/nnm-2018-0163 30088440 Gambaryan P.Y.; Kondrasheva I.G.; Severin E.S.; Guseva A.A.; Kamensky A.A.; Increasing the effciency of Parkinson’s Disease Treatment Using a poly(lactic-co-glycolic acid) (PLGA) based L-DOPA delivery system. Exp Neurobiol 2014,23(3),246-252 10.5607/en.2014.23.3.246 25258572 Pahuja R.; Seth K.; Shukla A.; Shukla R.K.; Bhatnagar P.; Chauhan L.K.S.; Saxena P.N.; Arun J.; Chaudhari B.P.; Patel D.K.; Singh S.P.; Shukla R.; Khanna V.K.; Kumar P.; Chaturvedi R.K.; Gupta K.C.; Trans-blood brain barrier delivery of dopamine-loaded nanoparticles reverses functional deficits in parkinsonian rats. ACS Nano 2015,9(5),4850-4871 10.1021/nn506408v 25825926 Nazem A.; Mansoori G.A.; Nanotechnology solutions for Alzheimer’s disease: advances in research tools, diagnostic methods and therapeutic agents. J Alzheimers Dis 2008,13(2),199-223 10.3233/JAD-2008-13210 18376062 Olivares D.; Deshpande V.K.; Shi Y.; Lahiri D.K.; Greig N.H.; Rogers J.T.; Huang X.; N-methyl D-aspartate (NMDA) receptor antagonists and memantine treatment for Alzheimer’s disease, vascular dementia and Parkinson’s disease. Curr Alzheimer Res 2012,9(6),746-758 10.2174/156720512801322564 21875407 Gong C.X.; Grundke-Iqbal I.; Iqbal K.; Targeting tau protein in Alzheimer’s disease. Drugs Aging 2010,27(5),351-365 10.2165/11536110-000000000-00000 20450234 Cummings J.L.; Doody R.; Clark C.; Disease-modifying therapies for Alzheimer disease. Neurology 2007,69(16),1622-1634 10.1212/01.wnl.0000295996.54210.69 17938373 Jakob-Roetne R.; Jacobsen H.; Alzheimer’s disease: From pathology to therapeutic approaches. Angew Chem Int Ed 2009,48(17),3030-3059 10.1002/anie.200802808 19330877 ManafiRad A.; Farzadfar F.; Habibi L.; Azhdarzadeh M.; Aghaverdi H.; Tehrani K.H.; Lotfi M.; Kehoe P.G.; Sheidaei A.; Ghasemian A.; Darzi E.R.; Mahmoodi R.; Mahmoudi M.; Is amyloid-β an innocent bystander and marker in Alzheimer’s disease? Is the liability of multivalent cation homeostasis and its influence on amyloid-β function the real mechanism? J Alzheimers Dis 2014,42(1),69-85 10.3233/JAD-140321 24787921 Rogawski M.A.; Convection-enhanced delivery in the treatment of epilepsy. Neurotherapeutics 2009,6(2),344-351 10.1016/j.nurt.2009.01.017 19332329 Shrivastava A.; Goyal M.K.; Gupta J.K.; Epileptogenic drugs and seizures: A comprehensive review of current knowledge Int. J Pharm Res 2020 10.31838/ijpr/2020.SP2.011 Pathan S.A.; Jain G.K.; Akhter S.; Vohora D.; Ahmad F.J.; Khar R.K.; Insights into the novel three ‘D’s of epilepsy treatment: drugs, delivery systems and devices. Drug Discov Today 2010,15(17-18),717-732 10.1016/j.drudis.2010.06.014 20603226 Kanakia S.; Toussaint J.D.; Mullick Chowdhury S.; Tembulkar T.; Lee S.; Jiang Y.P.; Lin R.Z.; Shroyer K.R.; Moore W.; Sitharaman B.; Dose ranging, expanded acute toxicity and safety pharmacology studies for intravenously administered functionalized graphene nanoparticle formulations. Biomaterials 2014,35(25),7022-7031 10.1016/j.biomaterials.2014.04.066 24854092 Daniel M.C.; Astruc D.; Gold nanoparticles: Assembly, supramolecular chemistry, quantum-size-related properties, and applications toward biology, catalysis, and nanotechnology. Chem Rev 2004,104(1),293-346 10.1021/cr030698+ 14719978 Khazipov R.; Khalilov I.; Tyzio R.; Morozova E.; Ben-Ari Y.; Holmes G.L.; Developmental changes in GABAergic actions and seizure susceptibility in the rat hippocampus. Eur J Neurosci 2004,19(3),590-600 10.1111/j.0953-816X.2003.03152.x 14984409

Rights & Permissions Print Cite
Article Metrics
34
1
Wayfinder Image
© 2024 Bentham Science Publishers | Privacy Policy