相关实验视频

Updated: Jan 8, 2026

Chemical Triphosphorylation of Oligonucleotides

Chemical Triphosphorylation of Oligonucleotides

Published on: June 2, 2022

使用简单酸盐捐赠体的关键RNA治疗构建块的酶合成

Qinglong Meng¹, Caecilie Benckendorff², Charlotte Morrill¹

¹Manchester Institute of Biotechnology and Department of Chemistry, University of Manchester, Manchester, UK.

Nature communications

|December 16, 2025

概括

这项研究提出了一种新的生物催化方法,用于生产RNA治疗中必不可少的修饰核酸三酸盐 (NTP). 工程酶有效地将核化物转化为关键构建块,简化制造和降低成本.

科学领域:

生物技术是生物技术.
合成生物学合成生物学
RNA疗法制造业制造业制造业

背景情况:

RNA疗法需要高效,可扩展和可持续的制造方法.
核三酸盐 (NTP) 是关键的构建块,特别是用于治疗应用的修改后的NTP.
目前的NTP生产方法通常依赖于昂贵的试剂和复杂的净化.

研究的目的:

开发一种新的生物催化方法,用于生产改性核三酸盐 (NTP).
设计酶以有效地将核化物转化为药学相关的NTP.
为了证明生产的NTP在寡核酸合成中的直接使用,绕过净化.

主要方法:

工程酸酸酶,聚酸酶和酸酶被利用.
开发了一种生物催化策略,使用廉价的酸盐供体将核化物转化为NTP.
该方法避免使用ATP作为酸盐供体,简化了下游加工.

主要成果:

成功生产了修改后的NTP,包括2'-O-甲氧乙烯-ATP (2'-MOE-ATP) 和2'--ATP.
证明了在酶性寡核酸合成中直接使用原始NTP的能力.
使用廉价的酸盐供体实现了高效的转化,降低了制造复杂性.

结论:

开发的生物催化方法提供了一种可持续且具有成本效益的方法来生产修改后的NTP.
这一策略大大简化了RNA治疗构件的制造过程.
在寡核酸合成中直接使用原始NTPs在RNA治疗生产方面取得了重大进展.

更多相关视频

Nucleoside Triphosphates - From Synthesis to Biochemical Characterization

Nucleoside Triphosphates - From Synthesis to Biochemical Characterization

Published on: April 3, 2014

Protocol for the Solid-phase Synthesis of Oligomers of RNA Containing a 2'-O-thiophenylmethyl Modification and Characterization via Circular Dichroism

Protocol for the Solid-phase Synthesis of Oligomers of RNA Containing a 2'-O-thiophenylmethyl Modification and Characterization via Circular Dichroism

Published on: July 28, 2017

相关概念视频

Biosynthesis of Nucleic Acids

Biosynthesis of Nucleic Acids

Nucleic acid biosynthesis is a fundamental biochemical process that produces the purine and pyrimidine nucleotides essential for DNA and RNA synthesis. This pathway maintains a balanced nucleotide pool, preventing imbalances that could jeopardize genetic integrity and cellular function. Given the crucial role of nucleotides, their synthesis is tightly regulated to ensure proper cellular homeostasis.Purine BiosynthesisThe biosynthesis of purine nucleotides begins with ribose-5-phosphate, a...

ATP and Macromolecule Synthesis

ATP and Macromolecule Synthesis

Biological macromolecules are organic compounds, predominantly composed of carbon atoms. The carbon atoms are covalently bonded with hydrogen, oxygen, nitrogen, and other minor elements. There are four major biological macromolecule classes: carbohydrates, lipids, proteins, and nucleic acids.
Most macromolecules are composed of single subunits, or building blocks, called monomers. The monomers combine with each other using covalent bonds to form larger molecules known as polymers.
Conversion of...

RNA Structure

RNA Structure

The basic structure of RNA consists of a string of ribonucleotides attached by phosphodiester bonds. Although most RNA is single-stranded, it can form complex secondary and tertiary structures. Such structures play essential roles in the regulation of transcription and translation.
Different Types of RNA Have the Same Basic Structure
There are three main types of ribonucleic acid (RNA) involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). All three...

RNA Structure

RNA Structure

Overview
The basic structure of RNA consists of a five-carbon sugar and one of four nitrogenous bases. Although most RNA is single-stranded, it can form complex secondary and tertiary structures. Such structures play essential roles in the regulation of transcription and translation.
Different Types of RNA Have the Same Basic Structure
There are three main types of ribonucleic acid (RNA): messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). All three RNA types consist of a...

Ribosomal RNA Synthesis

Ribosomal RNA Synthesis

Ribosome synthesis is a highly complex and coordinated process involving more than 200 assembly factors. The synthesis and processing of ribosomal components occurs not only in the nucleolus but also in the nucleoplasm and the cytoplasm of eukaryotic cells.
Ribosome biogenesis begins with the synthesis of 5S and 45S pre-rRNAs by distinct RNA polymerases. The primary transcripts are extensively processed and modified before they are bound and folded by ribosomal proteins and assembly factors,...

Eukaryotic RNA Polymerases

Eukaryotic RNA Polymerases

RNA Polymerase (RNAP) is conserved in all animals, with bacterial, archaeal, and eukaryotic RNAPs sharing significant sequence, structural, and functional similarities. Among the three eukaryotic RNAPs, RNA Polymerase II is most similar to bacterial RNAP in terms of both structural organization and folding topologies of the enzyme subunits. However, these similarities are not reflected in their mechanism of action.
All three eukaryotic RNAPs require specific transcription factors, of which the...