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Related Concept Videos

Experimental RNAi02:15

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...
siRNA - Small Interfering RNAs02:30

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...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
RNA Interference01:23

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 Interference01:23

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.
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In Vitro Selection of Engineered Transcriptional Repressors for Targeted Epigenetic Silencing
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Published on: May 5, 2023

Conditional RNAi: towards a silent gene therapy.

Sang-Kyung Lee1, Priti Kumar

  • 1Department of Bioengineering, Hanyang University, Seoul 133-797, South Korea.

Advanced Drug Delivery Reviews
|April 28, 2009
PubMed
Summary
This summary is machine-generated.

Conditional RNA interference (RNAi) allows external regulation of gene silencing, crucial for essential genes. This review explores methods for regulatable RNAi in mammals, focusing on therapeutic applications.

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • RNA interference (RNAi) offers broad gene downregulation capabilities.
  • Transgenic RNAi provides stable inheritance but its dominant nature restricts applications, especially for essential genes.

Purpose of the Study:

  • To review methods for externally regulatable RNA interference (conditional RNAi) in mammalian systems.
  • To highlight studies targeting therapeutically relevant molecules using these approaches.

Main Methods:

  • Overview of various conditional RNAi strategies in mammalian models.
  • Analysis of advantages and disadvantages of different approaches.
  • Discussion of suitability for RNAi-based gene therapy.

Main Results:

  • Conditional RNAi overcomes limitations of constitutive RNAi for essential genes.
  • Several externally regulatable systems have been developed and applied.
  • Specific therapeutic targets have been successfully modulated.

Conclusions:

  • Externally regulatable RNAi is essential for safe and effective gene silencing in mammalian systems.
  • The choice of conditional RNAi approach depends on the specific application and therapeutic goals.
  • Further development is needed to optimize RNAi-based gene therapy.