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Related Concept Videos

Delivery Pathways to the Lysosome01:36

Delivery Pathways to the Lysosome

Eukaryotic cells use different mechanisms to eliminate toxic waste obsolete and worn-out substances. Lysosomes play a pivotal role in this, and hence, these substances are carried to the lysosome from other parts of the cell and extracellular space through different pathways. The most elaborately studied pathways to the lysosome are the endocytic pathways.
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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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The Replisome03:01

The Replisome

DNA replication is carried out by a large complex of proteins that act in a coordinated matter to achieve high-fidelity DNA replication. Together this complex is known as the DNA replication machinery or the replisome.
The synthesis of the leading and lagging strands is a highly coordinated process. To explain this, the “Trombone model” was proposed by Bruce Alberts in 1980. The DNA loop formation starts when a primer is synthesized on the parent lagging strand. The loop grows with the...

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DNAzyme 10-23 - Based Nanomachines for Nucleic Acid Recognition
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DNAzyme 10-23 - Based Nanomachines for Nucleic Acid Recognition

Published on: February 9, 2024

DNAzyme delivery systems: getting past first base.

Mei Lin Tan1, Peter F M Choong, Crispin R Dass

  • 1University of Melbourne, Melbourne, Australia.

Expert Opinion on Drug Delivery
|February 26, 2009
PubMed
Summary
This summary is machine-generated.

DNAzyme technology shows promise for treating cancer and atherosclerosis, but effective drug delivery systems (DDSs) are crucial for clinical success. Enhancing DDS efficacy and safety is key for DNAzymes to become viable drug candidates.

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Drug Delivery Systems

Background:

  • DNAzyme technology offers potential therapeutic applications for diseases like cancer and atherosclerosis.
  • Current DNAzyme delivery relies on suboptimal systems, hindering clinical translation.
  • Existing drug delivery systems (DDSs) for gene knockdown agents, like antisense oligonucleotides, have faced challenges in clinical development.

Purpose of the Study:

  • To highlight the critical role of advanced drug delivery systems (DDSs) in realizing the therapeutic potential of DNAzymes.
  • To emphasize the need for improved efficacy, safety, and pharmacokinetic profiling of DNAzyme-based DDSs.
  • To address the challenges in translating DNAzyme technology into clinical applications by improving DDS implementation.

Main Methods:

  • Review of current DNAzyme technology and its therapeutic applications.
  • Analysis of existing drug delivery systems (DDSs) used for DNAzymes and related gene knockdown agents.
  • Evaluation of the limitations and requirements for successful clinical translation of DNAzyme-based therapies.

Main Results:

  • DNAzymes demonstrate potential as therapeutic agents but are hampered by inefficient drug delivery systems (DDSs).
  • While some DDSs like chitosan and polyethylenimine show promise, their efficacy, safety, and pharmacokinetics require further investigation.
  • The clinical success of gene knockdown agents like antisense oligonucleotides has been limited, underscoring the challenges in DDS development.

Conclusions:

  • The advancement of DNAzyme technology as a clinical therapeutic hinges on the development of superior drug delivery systems (DDSs).
  • Further research must focus on optimizing DDS efficacy, safety, and pharmacokinetic properties for DNAzymes.
  • Overcoming DDS limitations is essential for DNAzymes to achieve comparable status to other gene knockdown agents in clinical practice.