Services

Online Inquiry

Your Name

Phone Number

* E-mail Address

* Services Interested

Project Description

Please note that we are not a pharmacy or clinic, so we are unable to see patients and do not offer diagnostic and treatment services for individuals.

miRNA Modification Service

MicroRNAs (miRNAs) are pivotal regulators of gene expression, offering immense potential for therapeutic interventions in diseases ranging from cancer to neurodegenerative disorders. At Protheragen BioNucleics, we specialize in miRNA modification services, leveraging our expertise in oligonucleotide drug development to enhance stability, specificity, and delivery efficiency. As a leader in Oligonucleotide Drugs CRO and Oligonucleotide Modification Services, we bridge the gap between discovery and preclinical application through tailored chemical and structural optimizations.

Overview of miRNA Modification Services

miRNA therapeutics face challenges such as rapid degradation, off-target effects, and poor cellular uptake. Our services address these limitations by employing systematic chemical modifications to improve pharmacokinetics and pharmacodynamics. We focus on three core areas: sugar moiety modifications, nucleotide base alterations, and backbone engineering, each designed to refine miRNA interactions with biological systems. By integrating proprietary technologies, we deliver molecules with enhanced nuclease resistance, target affinity, and tissue-specific delivery, enabling robust in vivo performance.

Our Services

miRNA Sugar Modification

Modifying the ribose sugar (e.g., 2'-O-methyl, 2'-fluoro) enhances miRNA stability against nucleases and reduces immune activation. Our platform optimizes substitution patterns to balance efficacy and toxicity.

miRNA Nucleotide Base Modification

Incorporating non-canonical bases (e.g., locked nucleic acids, 5-methylcytosine) improves binding affinity to target mRNAs and mitigates off-target interactions. We tailor base edits to align with sequence-specific requirements.

miRNA Backbone Modification

Phosphorothioate or morpholino backbone substitutions increase serum stability and facilitate cellular uptake. We engineer backbone architectures to support sustained activity in complex biological environments.

Core Technologies Driving miRNA Innovation

Proprietary Chemical Modification Platform

Our platform integrates high-throughput screening and computational modeling to identify optimal modification sites. Using quantum mechanical simulations, we predict how chemical changes affect miRNA folding, hybridization, and interaction with RNA-induced silencing complexes (RISCs). This enables precision editing of nucleotides while preserving functional motifs.

Advanced Delivery Systems

We employ lipid nanoparticles (LNPs), GalNAc conjugates, and exosome-based carriers to ensure tissue-specific miRNA delivery. Our LNPs are optimized for endosomal escape, while GalNAc conjugates target hepatocytes via asialoglycoprotein receptors. Exosome engineering leverages endogenous vesicle trafficking for CNS and tumor penetration.

Bioinformatics-Driven Design

Machine learning algorithms analyze miRNA-mRNA interaction networks to predict off-target effects and optimize sequence specificity. Our tools evaluate thermodynamic stability, seed region complementarity, and cross-species conservation, ensuring therapeutic candidates meet stringent safety criteria.

cGMP-Compliant Synthesis and Purification

Scalable solid-phase synthesis produces modified miRNAs with >98% purity. We utilize HPLC and mass spectrometry for quality control, adhering to regulatory standards for preclinical-grade manufacturing.

In Vitro/ In Vivo Validation

Comprehensive tests are as follows. These technologies collectively ensure that our modified miRNAs achieve target engagement with minimal side effects.

Stability assays (serum/nuclease resistance).
Binding affinity measurements (surface plasmon resonance).
Functional studies (luciferase reporter, qPCR).
Toxicology profiling (cytokine release, immunogenicity).

Frequently Asked Questions

Q1: How do sugar modifications improve miRNA performance?

Sugar substitutions like 2'-O-methyl or 2'-fluoro shield miRNAs from ribonuclease degradation and reduce Toll-like receptor activation, extending half-life and improving safety profiles.

Q2: Can modified miRNAs target specific tissues?

Yes. Through GalNAc conjugation or LNP formulations, we achieve liver-, tumor-, or neuron-specific delivery, enhancing therapeutic precision.

Applications of Modified miRNAs

Oncology

Silencing oncogenic miRNAs or restoring tumor suppressors.

Neurodegenerative Diseases

Targeting mRNA aggregates in Alzheimer's or ALS.

Cardiovascular Disorders

Regulating pathways in atherosclerosis.

Rare Genetic Diseases

Correcting aberrant splicing or gene expression.

Our services and products are exclusively for authorized organizations in research, development, or manufacturing and are not intended for direct use by individuals or patients or as medical advice, diagnosis, or treatment.