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Related Concept Videos

Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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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.
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The first human genome sequencing project cost $2.7 billion and was declared complete in 2003, after 15 years of international cooperation and collaboration between several research teams and funding agencies. Today, with the advent of next-generation sequencing technologies, the cost and time of sequencing a human genome have dropped over 100 fold.
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DNA Microarrays02:34

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Microarrays are high-throughput and relatively inexpensive assays that can be automated to analyze large quantities of data at a time. They are used in genome-wide studies to compare gene or protein expression under two varied conditions, such as healthy and diseased states. Microarrays consist of glass or silica slides on which probe molecules are covalently attached through surface functionalization. Most commonly, the slides are prepared through the chemisorption of silanes to silica...
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DNA sequencing is a fundamental technique that is routinely used in the biological sciences. This method can be applied to a range of questions at different scales - from the sequencing of a cloned DNA fragment or the study of a mutation in a gene up to whole-genome sequencing. However, despite the widespread use of sequencing today, it was not until 1977 that Fredrick Sanger and his collaborators developed the chain-termination method to decode DNA sequences. It relies on the separation of a...
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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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Updated: Nov 5, 2025

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
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Smart Nucleic Acids as Future Therapeutics.

Jiahui Zhang1, Khalid Salaita2

  • 1Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University, Atlanta, GA, USA.

Trends in Biotechnology
|May 13, 2021
PubMed
Summary
This summary is machine-generated.

Smart nucleic acid therapeutics (NATs) offer new ways to treat diseases by responding to specific molecular triggers. This enhances drug safety and efficacy by activating treatments only where needed.

Keywords:
DNA nanostructuresnucleic acid therapeuticsphotochemical controlribodevicesspecificitystrand hybridization/displacement

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Therapeutics

Background:

  • Nucleic acid therapeutics (NATs) represent a significant advancement in medicine, offering potential treatments for previously undruggable diseases.
  • Despite progress, enhancing the specificity of NATs remains a critical challenge for broader clinical application.
  • Targeted activation of NATs within diseased cells is essential for improving safety and expanding therapeutic indications.

Purpose of the Study:

  • To review programmable modalities for developing "smart" nucleic acid therapeutics.
  • To explore responsive behaviors in NATs triggered by specific molecular inputs.
  • To summarize advancements in NATs designed for enhanced cellular specificity.

Main Methods:

  • Review of literature on smart NATs and responsive drug delivery systems.
  • Analysis of various input triggers (photo-uncaging, molecular signals) for NAT activation.
  • Examination of transduction mechanisms and output response functions in smart NATs.

Main Results:

  • Smart NATs can be programmed with responsive behaviors using diverse triggers.
  • Photo-uncaging and specific molecular inputs (transcripts, proteins, small molecules) enhance NAT specificity.
  • These strategies complement traditional ligand-based targeting for improved precision.

Conclusions:

  • Programmable modalities are key to developing sophisticated "smart" NATs.
  • Responsive NATs promise to increase therapeutic safety and broaden clinical applications.
  • Future research should focus on optimizing input-transduction-output systems for targeted therapies.