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Videos de Conceptos Relacionados

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...
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...
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.
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...
piRNA - Piwi-interacting RNAs02:57

piRNA - Piwi-interacting RNAs

PIWI-interacting RNAs, or piRNAs, are the most abundant short non-coding RNAs. More than 20,000 genes have been found in humans that code for piRNAs while only 2000 genes have been found for miRNAs. piRNAs can act at the transcriptional and post-transcriptional levels and have a vital role in silencing transposable elements present in germ cells. They are also involved in epigenetic silencing and activation. Previously, they were thought to function only in germ cells but new evidence suggests...
lncRNA - Long Non-coding RNAs02:39

lncRNA - Long Non-coding RNAs

In humans, more than 80% of the genome gets transcribed. However, only around 2% of the genome codes for proteins. The remaining part produces non-coding RNAs which includes ribosomal RNAs, transfer RNAs, telomerase RNAs, and regulatory RNAs, among other types. A large number of regulatory non-coding RNAs have been classified into two groups depending upon their length – small non-coding RNAs, such as microRNA, which are less than 200 nucleotides in length, and long non-coding RNA (lncRNA)...

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Rearing and Double-stranded RNA-mediated Gene Knockdown in the Hide Beetle, Dermestes maculatus
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Published on: December 28, 2016

Los siRNA de una sola cadena activan el ARN i en animales.

Walt F Lima1, Thazha P Prakash, Heather M Murray

  • 1Core Antisense Research, Isis Pharmaceuticals Inc., Carlsbad, CA 92010, USA.

Cell
|September 4, 2012
PubMed
Resumen

Los potentes siRNAs de una sola cadena (ss-siRNAs) pueden silenciar los genes in vivo sin una entrega compleja. Este descubrimiento simplifica la terapia de interferencia de ARN (RNAi) y ofrece nuevos conocimientos sobre los mecanismos de RNAi.

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Last Updated: May 19, 2026

Rearing and Double-stranded RNA-mediated Gene Knockdown in the Hide Beetle, Dermestes maculatus
09:57

Rearing and Double-stranded RNA-mediated Gene Knockdown in the Hide Beetle, Dermestes maculatus

Published on: December 28, 2016

Cell Based Assays of SINEUP Non-coding RNAs That Can Specifically Enhance mRNA Translation
10:21

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

Published on: February 1, 2019

Área de la Ciencia:

  • La bioquímica es la bioquímica.
  • Biología Molecular Biología Molecular
  • Farmacología Farmacología.

Sus antecedentes:

  • Las aplicaciones terapéuticas de pequeños ARN interferentes (siRNA) se ven obstaculizadas por la necesidad de sistemas de administración complejos.
  • La identificación de potentes ARN monocatenarios podría agilizar el desarrollo de fármacos basados en ARN.

Objetivo del estudio:

  • Desarrollar y caracterizar siRNAs de una sola cadena (ss-siRNAs) capaces de silenciar genes in vivo sin formulaciones de lípidos.
  • Explorar las relaciones estructura-actividad de ss-siRNAs para mejorar la potencia, la estabilidad y las propiedades farmacocinéticas.

Principales métodos:

  • Diseño iterativo y modificación química de ARN de una sola cadena.
  • Estudios de relación estructura-actividad que correlacionan las modificaciones químicas con la actividad de Argonaute 2 (AGO2), la potencia celular, la estabilidad de la nucleasa y la farmacocinética.
  • Pruebas in vivo de ss-siRNAs para la eficacia del silenciamiento de genes.

Principales resultados:

  • Se identificaron ss-siRNAs efectivos que silencian la expresión génica en animales sin entrega basada en lípidos.
  • Se demostró que la hebra pasajera no es esencial para el potente silenciamiento de genes a través de la vía de interferencia de ARN (RNAi).
  • Se confirmó que la actividad de ss-siRNA in vivo requiere un fosfato 5', lo que lleva al desarrollo de un análogo estable de 5'-(E) -vinilfosfonato (5'-VP).

Conclusiones:

  • Los potentes ss-siRNAs representan un enfoque simplificado para las terapias de RNAi, evitando los complejos requisitos de entrega.
  • Los hallazgos proporcionan una nueva perspectiva sobre el mecanismo de RNAi, destacando el papel esencial de la hebra guía y el fosfato 5'.
  • Este trabajo amplía las opciones para el desarrollo de agentes farmacológicos basados en ARNi.