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https://hdl.handle.net/10316/106220
Título: | Lysosomal Storage Disease-Associated Neuropathy: Targeting Stable Nucleic Acid Lipid Particle (SNALP)-Formulated siRNAs to the Brain as a Therapeutic Approach | Autor: | Coutinho, Maria Francisca Santos, Juliana Inês Mendonça, Liliana Matos, Liliana Prata, Maria João Jurado, Amália Lima, Maria C. Pedroso de Alves, Sandra |
Palavras-chave: | lysosomal storage diseases (LSDs); neuropathy; substrate reduction therapy (SRT); RNA interference (RNAi); siRNA nanodelivery systems; stable nucleic acid lipid particles (SNALPs) | Data: | 10-Ago-2020 | Editora: | MDPI | Título da revista, periódico, livro ou evento: | International Journal of Molecular Sciences | Volume: | 21 | Número: | 16 | Resumo: | 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. | URI: | https://hdl.handle.net/10316/106220 | ISSN: | 1422-0067 | DOI: | 10.3390/ijms21165732 | Direitos: | openAccess |
Aparece nas coleções: | I&D CNC - Artigos em Revistas Internacionais I&D CIBB - Artigos em Revistas Internacionais |
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