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Nucleic Acids as Therapeutic Targets and Agents
Published in David E. Thurston, Ilona Pysz, Chemistry and Pharmacology of Anticancer Drugs, 2021
RNA Interference (RNAi) is a natural process that occurs in all cells and is thought to have evolved as a protection mechanism against RNA viruses. It may also play a role in RNAi-related pathways such as shaping the chromatin structure of a genome. The cell responds to the introduction of extraneous dsRNA by destroying all intracellular mRNA of the same sequence. The phenomenon was first observed in the Caenorhabditis elegans worm and later in drosophila, trypanosomes, and planaria. The post-transcriptional gene silencing (PTGS) observed in plants is thought to operate through a similar RNAi mechanism.
Carriers for Nucleic Acid Delivery to the Brain
Published in Carla Vitorino, Andreia Jorge, Alberto Pais, Nanoparticles for Brain Drug Delivery, 2021
Apart from addressing challenges in nucleic acid delivery, the identification and selection of promising therapeutic genes or target genes for RNAi is of utmost importance and may include individualised therapies due to the recent tremendous progress in molecular profiling of patients [99]. Stability, specificity and immunogenicity of therapeutic nucleic acids can be optimised with advanced chemical modifications [124].
Introduction to Oral and Craniofacial Tissue Engineering
Published in Vincenzo Guarino, Marco Antonio Alvarez-Pérez, Current Advances in Oral and Craniofacial Tissue Engineering, 2020
María Verónica Cuevas González, Eduardo Villarreal-Ramírez, Adriana Pérez-Soria, Pedro Alberto López Reynoso, Vincenzo Guarino, Marco Antonio Alvarez-Pérez
The RNAi system has been considered a therapeutic promise in multiple fields of medicine, however, it is a system composed of various components controlling their functioning at the molecular level, its use in humans has been limited to animal models. Recently in 2018 the FDA approved the use of patisiran (Onpattro; Alnylam Pharmaceuticals), a siRNA that acts on the liver for the treatment of hATTR (hereditary transthyretin amyloidosis with polyneuropathy) (Setten et al. 2019).
RNAi therapeutics for diseases involving protein aggregation: fazirsiran for alpha-1 antitrypsin deficiency-associated liver disease
Published in Expert Opinion on Investigational Drugs, 2023
Pavel Strnad, Javier San Martin
The next frontier for this therapeutic approach might be the use of RNAi outside the liver, where there are many more possible targets to evaluate and diseases to treat. This is the next horizon in siRNA therapeutics development. The siRNA field has already begun to shift in that direction, with many companies trying to optimize delivery systems and find appropriate protein targets in muscle (e.g. facioscapulohumeral muscular dystrophy, muscular dystrophy), CNS (amyotrophic lateral sclerosis, Alzheimer’s disease), eye, and lung. An even more challenging, but highly attractive goal is to reach cancer tissues. The science must continue to evolve to address many more diseases in a targeted, specific, and reliable way. RNAi therapeutics are proving to be a desirable class of molecules capable of becoming these desperately needed medicines and satisfying the current unmet need for numerous intractable diseases.
Hyaluronic acid-modified redox-sensitive hybrid nanocomplex loading with siRNA for non-small-cell lung carcinoma therapy
Published in Drug Delivery, 2022
Daoyuan Chen, Peng Zhang, Minghui Li, Congcong Li, Xiaoyan Lu, Yiying Sun, Kaoxiang Sun
Recently, RNA interference (RNAi) technology has become a promising strategy for the treatment of major diseases, such as cancer, cardiovascular diseases, neurodegenerative diseases, etc. (Napoli et al., 1990; Guo et al., 2010; Kapoor et al., 2012; Kim et al., 2016; Lee et al., 2016). However, due to the existence of abundant RNase and the stability problem of RNA, including degradation during transport and transfection, RNAi-based agents are easily inactivated during systemic circulation; on the other side, the safe and effective delivery of RNAi agents, such as siRNA to target cells and efficient release into cytoplasm, still remains major hurdle for the clinical application of therapeutic RNAs, including siRNAs (Verma & Somia, 1997). Therefore, it is critical to design and develop effective RNA delivery vehicles for the clinical trials of RNAi therapeutics.
Genotoxicity evaluation of self-assembled-micelle inhibitory RNA-targeting amphiregulin (SAMiRNA-AREG), a novel siRNA nanoparticle for the treatment of fibrotic disease
Published in Drug and Chemical Toxicology, 2022
Hyeon-Young Kim, Tae Rim Kim, Sung-Hwan Kim, In-Hyeon Kim, Youngho Ko, Sungil Yun, In-Chul Lee, Han-Oh Park, Jong-Choon Kim
RNA interference (RNAi), a conserved mechanism for gene silencing, is attractive therapeutic tool for treating various diseases, including cancer, viral infections, and genetic disorders (Bumcrot et al. 2006, DiFiglia et al. 2007, Tseng et al. 2009, Steinbach et al. 2012). However, clinical application of small interfering RNA (siRNA) for RNAi is still limited by poor stability and inefficient cellular uptake of siRNA (Lee et al. 2014). Moreover, it has been reported that siRNA cause undesirable side effects owing to the toxicities associated with the off-target effects by silencing unintended genes or nonspecific immune stimulatory effects (Robbins et al. 2009). Recently, researchers have attempted to overcome the issues associated with toxicities and inefficient cellular uptake and target-gene silencing of siRNA by incorporating structural or chemical modifications in these molecules (Jeong et al. 2009, Kanasty et al. 2013, Falsini et al. 2014).