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Related Concept Videos

Experimental RNAi

Experimental RNAi

RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...

RNA Interference

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...

RNA Interference

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...

Microorganisms in Medicine and Therapeutics

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.

siRNA - Small Interfering RNAs

siRNA - Small Interfering RNAs

Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the ATP-dependent...

Ribosome Profiling

Ribosome Profiling

Ribosome profiling or ribo-sequencing is a deep sequencing technique that produces a snapshot of active translation in a cell. It selectively sequences the mRNAs protected by ribosomes to get an insight into a cell’s translation landscape at any given point in time.
Applications of ribosome profiling
Ribosome profiling has many applications, including in vivo monitoring of translation inside a particular organ or tissue type and quantifying new protein synthesis levels.
The technique helps...

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Related Experiment Video

Updated: Jun 17, 2026

Cell Based Assays of SINEUP Non-coding RNAs That Can Specifically Enhance mRNA Translation

Cell Based Assays of SINEUP Non-coding RNAs That Can Specifically Enhance mRNA Translation

Published on: February 1, 2019

RNAi-based therapeutics-current status, challenges and prospects.

Katrin Tiemann¹, John J Rossi

¹Department of Molecular Biology, Beckman Research Institute of the City of Hope, Duarte, CA, USA.

EMBO Molecular Medicine

|January 6, 2010

Summary

RNA interference (RNAi) therapeutics offer targeted gene silencing for new treatments. Overcoming challenges like delivery and off-target effects is key to realizing their full therapeutic potential.

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Area of Science:

Biotechnology and Molecular Medicine
Gene Therapy and Therapeutics

Background:

RNA interference (RNAi) is a natural gene silencing mechanism utilizing small RNAs.
The therapeutic potential of RNAi was recognized shortly after its discovery, sparking rapid research and development.
RNAi-based drug development has progressed significantly, with multiple clinical trials underway.

Purpose of the Study:

To review the current landscape of RNAi-based therapeutics.
To identify and discuss the primary challenges hindering therapeutic applications of RNAi.
To explore strategies for overcoming these challenges to advance RNAi-based medicine.

Main Methods:

Review of existing literature on RNA interference mechanisms and therapeutic applications.
Analysis of current clinical trial data and research findings in the field of RNAi therapeutics.
Identification of key technical and biological hurdles in RNAi drug development.

Main Results:

RNAi therapeutics have shown promise and advanced to clinical trials.
Significant challenges remain, including off-target effects, immune responses, and efficient cellular delivery.
Ongoing research is focused on developing sophisticated delivery systems and improving specificity.

Conclusions:

RNAi therapeutics represent a promising new class of drugs with the potential for targeted gene inhibition.
Addressing challenges in delivery, specificity, and immunogenicity is crucial for successful clinical translation.
Continued innovation in delivery technologies and molecular design will be essential for the future of RNAi-based therapies.