[Therapeutic oligonucleotides: a review].

Area of Science:

• Biotechnology
• Molecular Biology
• Chemical Biology

Background:

• Oligonucleotides are crucial for gene regulation and targeted gene therapy, applicable to viral, tumor, and hereditary diseases.
• Therapeutic oligonucleotides encompass various types like antisense oligonucleotides and small interfering RNA (siRNA).
• Modifications enhance oligonucleotide stability and specificity, mitigating issues like poor in vivo stability and side effects inherent in natural forms.

Purpose of the Study:

• To review a novel thermocyclic reaction for oligonucleotide amplification.
• To introduce the Polymerase-endonuclease Amplification Reaction (PEAR) as a potential manufacturing method.
• To compare PEAR with traditional chemical synthesis for oligonucleotide production.

Main Methods:

• Review of a novel thermocyclic reaction: Polymerase-endonuclease Amplification Reaction (PEAR).
• PEAR utilizes two thermostable enzymes for oligonucleotide amplification.
• Comparison of PEAR with conventional chemical synthesis methods.

Main Results:

• PEAR is a simple, efficient, and stable method for oligonucleotide amplification.
• Enzymatic production via PEAR offers an alternative to chemical synthesis.
• PEAR shows potential for large-scale oligonucleotide production.

Conclusions:

• The Polymerase-endonuclease Amplification Reaction (PEAR) presents a robust enzymatic alternative for oligonucleotide synthesis.
• PEAR could enable cost-effective, large-scale manufacturing of therapeutic and non-therapeutic oligonucleotides.
• This method addresses limitations associated with traditional chemical synthesis, including purity and cost.