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Updated: Jul 7, 2026
09:09
Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery
Published on: May 2, 2019
Delivering silence: advancements in developing siRNA therapeutics
Tatiana I Novobrantseva1, Akin Akinc, Anna Borodovsky
1Alnylam Pharmaceuticals Inc, 300 Third Street, Cambridge, MA 02142, USA. tnovobrantseva@alnylam.com
Current Opinion in Drug Discovery & Development
|February 20, 2008
Summary
Small interfering RNA (siRNA) therapeutics are advancing rapidly into human clinical trials. This review covers recent in vivo data, delivery technologies, and ongoing clinical trials for siRNA drugs.
Area of Science:
- Biotechnology
- Pharmacology
- Molecular Biology
Background:
- Small interfering RNA (siRNA) therapeutics represent a promising class of drugs.
- The development of siRNA-based treatments is accelerating, with many candidates entering human clinical trials.
Purpose of the Study:
- To review recent in vivo data for siRNA therapeutics.
- To provide an overview of current in vivo delivery technologies for siRNA.
- To summarize ongoing clinical trials involving siRNA drugs.
Main Methods:
- Literature review of recent in vivo studies on siRNA therapeutics.
- Analysis of key in vivo delivery technologies.
- Compilation of data from ongoing clinical trials.
Main Results:
- The field of siRNA therapeutics is experiencing significant growth.
- Various in vivo delivery technologies are being utilized and developed.
- Numerous clinical trials are actively investigating the efficacy and safety of siRNA drugs.
Conclusions:
- siRNA therapeutics show substantial potential and are progressing through clinical development.
- Effective in vivo delivery remains a critical factor for therapeutic success.
- Ongoing clinical trials will provide crucial insights into the future of siRNA-based medicine.
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02:30siRNA - 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...
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...
02:15Experimental 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...
01:23RNA 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...
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01:23RNA 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...
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...
02:30Small interfering RNAs (siRNA)
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...
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...
01:22MicroRNAs
MicroRNA (miRNA) are short, regulatory RNA transcribed from introns (non-coding regions of a gene) or intergenic regions (stretches of DNA present between genes). Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself, forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA...