Exploring RNA therapeutics for urea cycle disorders
RNA has triggered a significant shift in modern medicine, providing a promis-ing way to revolutionize disease treatment methods. Different therapeutic RNA modalities have shown promise to replace, supplement, correct, suppress,or eliminate the expression of a targeted gene. Currently, there are 22 R...
| Autores: | , , |
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| Tipo de documento: | artigo |
| Data de publicação: | 2024 |
| País: | España |
| Recursos: | Universidad Autónoma de Madrid |
| Repositório: | Biblos-e Archivo. Repositorio Institucional de la UAM |
| Idioma: | inglês |
| OAI Identifier: | oai:repositorio.uam.es:10486/715935 |
| Acesso em linha: | http://hdl.handle.net/10486/715935 https://dx.doi.org/10.1002/jimd.12807 |
| Access Level: | Acceso aberto |
| Palavra-chave: | Antisense Oligonucleotides mRNA therapy Ornithine Transcarbamylase Deficiency Pseudoexon Splicing Urea Cycle Biología y Biomedicina / Biología |
| Resumo: | RNA has triggered a significant shift in modern medicine, providing a promis-ing way to revolutionize disease treatment methods. Different therapeutic RNA modalities have shown promise to replace, supplement, correct, suppress,or eliminate the expression of a targeted gene. Currently, there are 22 RNA-based drugs approved for clinical use, including the COVID-19 mRNA vac-cines, whose unprecedented worldwide success has meant a definitive boost inthe RNA research field. Urea cycle disorders (UCD), liver diseases with highmortality and morbidity, may benefit from the progress achieved, as differentgenetic payloads have been successfully targeted to liver using viral vectors, N-acetylgalactosamine (GalNAc) conjugations or lipid nanoparticles (LNP). This review explores the potential of RNA-based medicines for UCD and theongoing development of applications targeting specific gene defects, enzymes, or transporters taking part in the urea cycle. Notably, LNP-formulated mRNA therapy has been assayed preclinically for citrullinemia type I (CTLN1), adolescent and adult citrin deficiency, argininosuccinic aciduria, arginase defi-ciency and ornithine transcarbamylase deficiency, in the latter case hasprogressed to the clinical trials phase |
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