Please use this identifier to cite or link to this item:
https://hdl.handle.net/10316/106220
DC Field | Value | Language |
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dc.contributor.author | Coutinho, Maria Francisca | - |
dc.contributor.author | Santos, Juliana Inês | - |
dc.contributor.author | Mendonça, Liliana | - |
dc.contributor.author | Matos, Liliana | - |
dc.contributor.author | Prata, Maria João | - |
dc.contributor.author | Jurado, Amália | - |
dc.contributor.author | Lima, Maria C. Pedroso de | - |
dc.contributor.author | Alves, Sandra | - |
dc.date.accessioned | 2023-03-27T08:43:35Z | - |
dc.date.available | 2023-03-27T08:43:35Z | - |
dc.date.issued | 2020-08-10 | - |
dc.identifier.issn | 1422-0067 | pt |
dc.identifier.uri | https://hdl.handle.net/10316/106220 | - |
dc.description.abstract | More than two thirds of Lysosomal Storage Diseases (LSDs) present central nervous system involvement. Nevertheless, only one of the currently approved therapies has an impact on neuropathology. Therefore, alternative approaches are under development, either addressing the underlying enzymatic defect or its downstream consequences. Also under study is the possibility to block substrate accumulation upstream, by promoting a decrease of its synthesis. This concept is known as substrate reduction therapy and may be triggered by several molecules, such as small interfering RNAs (siRNAs). siRNAs promote RNA interference, a naturally occurring sequence-specific post-transcriptional gene-silencing mechanism, and may target virtually any gene of interest, inhibiting its expression. Still, naked siRNAs have limited cellular uptake, low biological stability, and unfavorable pharmacokinetics. Thus, their translation into clinics requires proper delivery methods. One promising platform is a special class of liposomes called stable nucleic acid lipid particles (SNALPs), which are characterized by high cargo encapsulation efficiency and may be engineered to promote targeted delivery to specific receptors. Here, we review the concept of SNALPs, presenting a series of examples on their efficacy as siRNA nanodelivery systems. By doing so, we hope to unveil the therapeutic potential of these nanosystems for targeted brain delivery of siRNAs in LSDs. | pt |
dc.description.sponsorship | This work was supported by the Portuguese Society for Metabolic Disorders (Sociedade Portuguesa de Doenças Metabólicas, SPDM—Bolsa SPDM de apoio à investigação Dr. Aguinaldo Cabral 2018; 2019DGH1629/ SPDM2018I&D) and Sanfilippo Children’s Foundation (SCF Incubator Grant 2019; 2019DGH1656/SCF2019I&D). Additional support came from the European Regional Development Fund (ERDF), through the Centro 2020 Regional Operational Programme (project CENTRO-01-0145-FEDER-000008: BrainHealth 2020), and theCOMPETE 2020—Operational Programme for Competitiveness and Internationalisation and Portuguese national funds via FCT—Fundação para a Ciência e a Tecnologia (projects POCI-01-0145-FEDER-016390: CANCEL STEM and POCI-01-0145-FEDER-007440; UIDB/04539/2020). L.S.M. is funded by FCT/MCTES National Funds under project CEECIND/04242/2017. | - |
dc.language.iso | eng | pt |
dc.publisher | MDPI | pt |
dc.rights | openAccess | pt |
dc.rights.uri | http://creativecommons.org/licenses/by/4.0/ | pt |
dc.subject | lysosomal storage diseases (LSDs) | pt |
dc.subject | neuropathy | pt |
dc.subject | substrate reduction therapy (SRT) | pt |
dc.subject | RNA interference (RNAi) | pt |
dc.subject | siRNA nanodelivery systems | pt |
dc.subject | stable nucleic acid lipid particles (SNALPs) | pt |
dc.subject.mesh | Animals | pt |
dc.subject.mesh | Brain | pt |
dc.subject.mesh | Central Nervous System Diseases | pt |
dc.subject.mesh | Drug Delivery Systems | pt |
dc.subject.mesh | Drug Stability | pt |
dc.subject.mesh | Humans | pt |
dc.subject.mesh | Liposomes | pt |
dc.subject.mesh | Lysosomal Storage Diseases | pt |
dc.subject.mesh | Nanoparticles | pt |
dc.subject.mesh | RNA Interference | pt |
dc.subject.mesh | RNA, Double-Stranded | pt |
dc.subject.mesh | RNA, Small Interfering | pt |
dc.title | Lysosomal Storage Disease-Associated Neuropathy: Targeting Stable Nucleic Acid Lipid Particle (SNALP)-Formulated siRNAs to the Brain as a Therapeutic Approach | pt |
dc.type | article | - |
degois.publication.firstPage | 5732 | pt |
degois.publication.issue | 16 | pt |
degois.publication.title | International Journal of Molecular Sciences | pt |
dc.peerreviewed | yes | pt |
dc.identifier.doi | 10.3390/ijms21165732 | pt |
degois.publication.volume | 21 | pt |
dc.date.embargo | 2020-08-10 | * |
uc.date.periodoEmbargo | 0 | pt |
item.grantfulltext | open | - |
item.fulltext | Com Texto completo | - |
item.openairetype | article | - |
item.languageiso639-1 | en | - |
item.openairecristype | http://purl.org/coar/resource_type/c_18cf | - |
item.cerifentitytype | Publications | - |
crisitem.author.researchunit | CNC - Center for Neuroscience and Cell Biology | - |
crisitem.author.researchunit | CNC - Center for Neuroscience and Cell Biology | - |
crisitem.author.researchunit | CNC - Center for Neuroscience and Cell Biology | - |
crisitem.author.orcid | 0000-0002-0218-9690 | - |
crisitem.author.orcid | 0000-0002-0583-1028 | - |
crisitem.author.orcid | 0000-0001-7095-5337 | - |
crisitem.author.orcid | 0000-0003-1844-5027 | - |
Appears in Collections: | I&D CNC - Artigos em Revistas Internacionais I&D CIBB - Artigos em Revistas Internacionais |
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