A carregar...
| Nome: | Descrição: | Tamanho: | Formato: | |
|---|---|---|---|---|
| 1.14 MB | Adobe PDF |
Orientador(es)
Resumo(s)
Retinal diseases lead to severe vision loss and are currently a major cause of vision impairment in industrialized countries. The significant number of genetic defects of the retina underlying these disorders, coupled to the absence of effective treatments, require new therapeutic solutions. Recent gene therapy developments in the field of ophthalmic research reveal the great potential of this approach. In recent years, non-viral vectors have been extensively studied due to their properties such as large gene packaging capacity and low immunogenicity. Hitherto, their development and optimisation for retinal gene therapy have been hindered by their inability to directly target retinal cells. The goal of this review is to summarize the most promising strategies to direct non-viral vectors for retinal cells to avoid off-target effects and promote their specific uptake, gene expression and overall efficiency.
Descrição
Funding Information: The authors acknowledge the financial support of Funda\u00E7\u00E3o para a Ci\u00EAncia e Tecnologia (PTDC/BTM/ORG/28121/2017 and EXPL/BTM-ORG/0500/2021 to GS). iNOVA4Health\u2014UID/Multi/04462/2020, a program financially supported by Funda\u00E7\u00E3o para a Ci\u00EAncia e Tecnologia/Minist\u00E9rio da Educa\u00E7\u00E3o e Ci\u00EAncia, through national funds and co-funded by FEDER under the PT2020 Partnership Agreement is also acknowledged. Publisher Copyright: Copyright © 2022 Bitoque, Fernandes, Oliveira and Silva.
Palavras-chave
cellular targeting gene therapy non-viral vectors polymers retina Clinical Biochemistry Medicine (miscellaneous) Pharmacology