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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...
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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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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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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...
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Targeted RNA Sequencing Assay to Characterize Gene Expression and Genomic Alterations
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Amplifying gene expression with RNA-targeted therapeutics.

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  • 1OPKO Health, Miami, FL, USA.

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New RNA-targeted therapeutics offer a novel approach to activate gene expression for treating diseases. These therapies focus on modulating non-coding RNA networks to increase protein production, overcoming limitations of current treatments.

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Area of Science:

  • Molecular Biology
  • Genetics
  • Drug Development

Background:

  • Many diseases stem from insufficient gene expression, necessitating methods to boost protein production.
  • Traditional drug development favors inhibitory effects, while protein replacement and gene therapy have limitations.
  • Non-coding RNAs (ncRNAs) represent extensive regulatory networks with potential for therapeutic intervention.

Purpose of the Study:

  • To review emerging RNA-targeted therapeutics for gene activation.
  • To highlight novel strategies for amplifying specific protein production.
  • To discuss the opportunities and challenges in translating these therapies to clinical practice.

Main Methods:

  • Exploration of RNA-targeting small molecules.
  • Investigation of nucleic acid-based therapeutic modalities.
  • Analysis of strategies targeting messenger RNA (mRNA) stability and ncRNA-mediated gene regulation.

Main Results:

  • Emerging RNA-targeted therapeutics offer precise modulation of gene expression.
  • These approaches can directly influence mRNA stability and ncRNA regulatory functions.
  • Development is progressing for novel therapeutic strategies beyond traditional methods.

Conclusions:

  • RNA-targeted therapeutics present a promising avenue for gene activation therapies.
  • Modulating ncRNA networks provides a new strategy to address diseases caused by insufficient protein expression.
  • Further research and development are crucial for clinical translation of these innovative treatments.