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Synthesis of new DNA molecules is carried out by the enzyme DNA polymerase, which adds nucleotides on the daughter strand complementary to the template DNA strand. DNA polymerase has a higher affinity to add the correct base and ensures fidelity during DNA replication. Furthermore,  it exhibits proofreading activity during replication, using an exonuclease domain that cuts off incorrect nucleotides from the nascent DNA strand.
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[Correcting pathogenic mutations using prime editing: an overview].

Camille Bouchard1, Kelly Godbout1, Jacques P Tremblay1

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Prime editing is a novel gene editing technology that precisely modifies DNA. This review covers its applications in disease modeling and gene therapy, highlighting delivery challenges for therapeutic use.

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Gene editing technologies are rapidly advancing.
  • Prime editing represents a significant innovation in precise DNA modification.
  • Current gene editing tools face limitations in accuracy and scope.

Purpose of the Study:

  • To review the latest advancements in Prime editing technology.
  • To explore the applications of Prime editing in creating disease models.
  • To discuss the potential of Prime editing in treating hereditary diseases.
  • To identify key challenges in delivering Prime editing therapeutics in vivo.

Main Methods:

  • Utilizes a Cas9 nickase fused to a reverse transcriptase.
  • Employs a prime editing guide RNA (pegRNA) for targeting and editing.
  • Reviews existing literature on Prime editing applications and delivery strategies.

Main Results:

  • Prime editing enables precise introduction of pathogenic mutations for disease modeling.
  • It offers potential for correcting disease-causing mutations in genetic disorders.
  • Significant challenges remain in achieving efficient and targeted delivery of Prime editing components to specific organs in vivo.

Conclusions:

  • Prime editing is a powerful tool for both basic research and therapeutic development.
  • Overcoming in vivo delivery hurdles is critical for realizing Prime editing's clinical potential.
  • Continued research is needed to optimize delivery methods and expand therapeutic applications.