RNA interference (RNAi) is a naturally occurring cellular post-transcriptional gene regulation process that utilizes small double-stranded RNAs to trigger and guide gene silencing. By introducing synthetic RNA duplexes called small-interfering RNAs (siRNAs), we can harness the RNAi machinery for therapeutic gene control and the treatment of various diseases.
Several viral epidemics – such as the epidemics caused by H1N1 influenza virus, human immunodeficiency virus (HIV), Ebola virus, Zika virus, severe acute respiratory syndrome (SARS) virus, Middle East respiratory syndrome (MERS) virus and SARS-CoV-2 – have profoundly impacted global human health. Early identification of infected and/or infectious persons and isolating them from the population are some of the most effective and evident measures to prevent human-to-human spreading.
The promise of RNA interference based therapeutics is made evident by the recent surge of biotechnological drug companies that pursue such therapies and their progression into human clinical trials. The present invention discloses novel RNA and RNA/DNA nanoparticles including multiple siRNAs, RNA aptamers, fluorescent dyes, and proteins. These RNA nanoparticles are useful for various nanotechnological applications.
RNA interference (RNAi) is a naturally occurring post-transcriptional gene regulation process that represses the expression of specific genes. Exploiting endogenous RNAi by externally-delivered small-interfering RNA (siRNA) is a promising therapeutic for the treatment of various diseases representing several major unmet medical needs.
Current treatments for cancer and viral infection are limited remedies that often suppress cell or viral replication rather than eliminate diseased cells entirely from the body. A further limitation is that these therapies often compromise healthy cells as well, leaving problems of recurrence and side effects.
Researchers at developed a novel therapeutic nanoparticle (NP) system harboring therapeutic small siRNA that can significantly enhance effectiveness and specificity of treatments by killing diseased cells.
Nanoparticles such as lipid-based nanoparticles (LNPs) represent a relatively new era of targeted drug delivery systems wherein these biocompatible particles can carry the drug(s) of interest to a specific tumor site. The new generation of nanoparticles, known as stealth nanoparticles, are engineered to have a coating of polyethylene glycol polymer (PEG) or other glycolipids that enable them to evade the immune system and have a longer circulation lifespan as well as improved bioavailability to diseased tissue and reduced non-specific toxicity.
The Corona virus disease, 2019 (COVID-19) pandemic is a worldwide public health crisis with over 153 million confirmed cases and 3.2 million deaths as of April 2021. COVID-19 is caused by a novel coronavirus called SARS-CoV-2. SARS-COV-2 infects hosts via its spike (S) protein, which has two portions, S1 that binds the cell and S2 that is involved in viral entry via fusion with the cell membrane. There are several vaccines available for COVID-19 patients that directly target SARS-CoV-2 by systemic immunization.
Multi-parameter flow cytometry has been extensively used in multiple disciplines of biological discoveries, including immunology and cancer research. However, the disadvantage of traditional flow cytometry platforms using excitation lasers and fluorescence detectors is spectral overlap when using multiple dyes on the same biological sample. Metaethical compensation of spectral overlap could only be effective to a certain degree. Mass cytometry is advantageous compared to flow cytometry but is pricey and requires highly skilled operators.
Human immunodeficiency virus (HIV) infections remain a pandemic, most prevalent in Africa and the Americas. Anti-retroviral treatments have been effective in preventing spread of the virus and active outbreaks of acquired immune deficiency syndrome (AIDS). However, the development and deployment of an effective vaccine would provide long-lasting protection and alleviate the need to depend heavily on prevention methods that require continued access and adherence.
This technology includes a straightforward and versatile nanotechnology-based approach for in situ therapeutic vaccination that exploits a primary tumor as a vaccine depot to initiate robust personalized anti-tumor immune responses.