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Reporter Genes02:11

Reporter Genes

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Reporter genes are a type of protein-coding gene that are often tagged to a gene of interest. Once inside a target cell, reporter genes usually produce visually identifiable characteristics like fluorescence and luminescence when expressed along with the gene of interest. Thus, reporter genes “report” the presence or absence of genes of interest in an organism, determine the gene expression pattern, or track the physical location of a DNA segment or protein in the cell.
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Regulation of Expression at Multiple Steps01:23

Regulation of Expression at Multiple Steps

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The gene expression in cells is regulated at different stages: (i) transcription, (ii) RNA processing, (iii) RNA localization, and (iv) translation. Transcriptional regulation is mediated by regulatory proteins such as transcription factors, activators, or repressors—these control gene expression by initiating or inhibiting the transcription of genes. Once a precursor or pre-mRNA is produced, it undergoes post-transcriptional modification, including 5' capping, splicing, and the...
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Regulation of Expression Occurs at Multiple Steps02:24

Regulation of Expression Occurs at Multiple Steps

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Gene expression can be regulated at almost every step from gene to protein. Transcription is the step that is most commonly regulated. This involves the binding of proteins to short regulatory sequences on the DNA. This association can either promote or inhibit the transcription of a gene associated with the respective sequence.
Transcription results in the generation of precursor (pre-mRNA) that consists of both exons and introns, which needs further processing before being translated to a...
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Conservative Site-specific Recombination and Phase Variation02:53

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Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...
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What is Gene Expression?01:42

What is Gene Expression?

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Overview
Gene expression is the process in which DNA directs the synthesis of functional products, that is, proteins. Cells can regulate gene expression at various stages. It allows organisms to generate different cell types and enables cells to adapt to internal and external factors.
Genetic Information Flows from DNA to RNA to Protein
A gene is a stretch of DNA that serves as the blueprint for functional RNAs and proteins. Since DNA is made up of nucleotides and proteins consist of amino...
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What is Gene Expression?01:36

What is Gene Expression?

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A gene is a stretch of DNA that serves as the blueprint for functional RNAs and proteins. Since DNA is comprised  of nucleotides and proteins are comprised of amino acids, a mediator is required to convert the information encoded in DNA into proteins. This mediator is the messenger RNA (mRNA). mRNA copies the blueprint from DNA by a process called transcription. In eukaryotes, transcription occurs in the nucleus by complementary base-pairing with the DNA template. The mRNA is then...
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Updated: Jan 13, 2026

In vivo Application of the REMOTE-control System for the Manipulation of Endogenous Gene Expression
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Expresión génica regulable in vivo mediante adaptámeros

Jack Bryant1, Laura Herron1, Yesh Doctor1

  • 1Department of Bioengineering, University of California San Diego, CA, USA.

bioRxiv : the preprint server for biology
|January 7, 2026
PubMed
Resumen
Este resumen es generado por máquina.

Los investigadores desarrollaron un interruptor de ARN compacto llamado adaptámero (aptámero modulable por ADAR) para un control preciso de la expresión génica. Este sistema permite una terapia génica regulable aprovechando moléculas pequeñas y la edición de ARN endógena, mostrando resultados prometedores en la reversión de la obesidad en ratones.

Palabras clave:
adaptámeroterapia génicaedición de ARNARN regulablemedicina genética

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Área de la Ciencia:

  • Biotecnología
  • Biología Molecular
  • Terapia Génica

Sus antecedentes:

  • Los sistemas actuales de expresión génica inducible para terapia génica enfrentan desafíos como el gran tamaño, la corta duración de la inducción y los reguladores inmunogénicos, lo que dificulta la aplicación in vivo.
  • Lograr un control preciso y reversible de la expresión de transgenes es fundamental para la seguridad y eficacia de las terapias génicas.

Objetivo del estudio:

  • Desarrollar un interruptor de ARN novedoso y compacto para el control preciso y reversible de la expresión de transgenes.
  • Crear un sistema compatible con la clínica para terapias génicas regulables utilizando la edición de ARN endógena.

Principales métodos:

  • Se diseñó un interruptor de ARN compacto (<120 pb) denominado adaptámero, que integra un aptámero sensible a moléculas pequeñas con la edición de ARN mediada por ADAR.
  • Se demostró la funcionalidad del adaptámero en varias líneas celulares, incluidas las células T humanas.
  • Se utilizó la administración de virus adenoasociados (AAV) en ratones para controlar la expresión de FGF21 a través del sistema adaptámero.

Principales resultados:

  • El sistema adaptámero permite la modulación precisa de la traducción de proteínas dependiente de moléculas pequeñas a través de un mecanismo postranscripcional.
  • Los adaptámeros mostraron una alta funcionalidad en múltiples líneas celulares, incluidas las células T humanas.
  • In vivo, la expresión de FGF21 controlada por adaptámero administrada por AAV condujo a una remodelación metabólica significativa, un aumento del gasto energético y la reversión de la obesidad en ratones.

Conclusiones:

  • El adaptámero representa un interruptor de ARN mínimo y programable para medicinas genéticas regulables.
  • Este sistema ofrece un enfoque compatible con la clínica para la implementación segura de proteínas pleiotrópicas y de dosis limitada en la terapia génica.