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Formulation approaches to short interfering RNA and MicroRNA: challenges and implications
Diana Guzman-Villanueva1, Ibrahim M El-Sherbiny, Dea Herrera-Ruiz
1Division of Pharmaceutics, College of Pharmacy, The University of Texas at Austin, Texas 78712-0120, USA.
Journal of Pharmaceutical Sciences
|August 29, 2012
Summary
RNA interference uses short interfering RNA (siRNA) and microRNA (miRNA) to silence disease-causing genes. Nonviral delivery strategies are crucial for overcoming barriers to effective in vivo gene therapy.
Area of Science:
- Biotechnology
- Molecular Biology
- Gene Therapy
Background:
- RNA interference (RNAi) offers therapeutic potential for genetic and acquired diseases.
- Delivery of small interfering RNA (siRNA) and microRNA (miRNA) is essential for gene silencing.
- Significant physicochemical and biological barriers impede efficient in vivo delivery of RNAi therapeutics.
Purpose of the Study:
- To review the challenges in siRNA and miRNA delivery for therapeutic applications.
- To discuss nonviral delivery strategies for overcoming these delivery barriers.
- To highlight technologies that have advanced to clinical trials for RNAi-based therapies.
Main Methods:
- Literature review of nonviral delivery strategies for siRNA and miRNA.
- Analysis of technologies progressing in clinical trials.
- Focus on both local and systemic delivery approaches.
Main Results:
- Nonviral vectors are key to overcoming biological and chemical barriers for RNAi delivery.
- Several nonviral delivery technologies have shown promise and entered clinical evaluation.
- Progress has been made in both localized and systemic administration of RNAi agents.
Conclusions:
- Effective in vivo delivery of siRNA and miRNA remains a critical challenge for clinical translation.
- Nonviral delivery systems represent a promising avenue for advancing RNAi-based therapies.
- Ongoing research and clinical trials are vital for realizing the full therapeutic potential of RNA interference.
Related Concept Videos
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...
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...
02:15Experimental 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...
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...
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...
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 ends...