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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 Backbone Modification

At Protheragen BioNucleics, we are dedicated to advancing the field of oligonucleotide therapeutics through our comprehensive Oligonucleotide Drugs CRO services. Specializing in innovative Oligonucleotide Modification Services, we offer cutting-edge solutions for miRNA-based therapies. Our expertise in miRNA Modification Services enables us to tailor miRNA molecules for enhanced stability, specificity, and therapeutic efficacy.

Overview of MicroRNAs (miRNAs) Backbone Modification

miRNAs are small, non-coding RNA molecules that play pivotal roles in regulating gene expression. Despite their therapeutic potential, native miRNAs face challenges such as rapid degradation by nucleases, poor cellular uptake, and off-target effects. Backbone modification of miRNAs involves altering the phosphate-deoxyribose backbone to improve their biophysical properties. These modifications enhance nuclease resistance, increase binding affinity to target mRNAs, and reduce immunogenicity, thereby optimizing miRNAs for preclinical applications.

Our miRNA Backbone Modification Services

At Protheragen BioNucleics, we provide a comprehensive suite of miRNA backbone modification services designed to meet the diverse needs of our clients:

Phosphorothioate (PTO) Modification

We introduce sulfur atoms into the miRNA backbone to replace non-bridging oxygen atoms in the phosphate group. This modification significantly enhances nuclease resistance and improves the pharmacokinetic profile of miRNAs without adversely affecting their biological activity.

Locked Nucleic Acid (LNA) Incorporation

We specialize in integrating LNA nucleotides into miRNA sequences. LNAs constrain the ribose ring, enhancing hybridization affinity and specificity toward target mRNAs. This results in potent gene silencing effects at lower dosages, minimizing potential off-target interactions.

Peptide Nucleic Acid (PNA) Synthesis

Our experts synthesize PNA-modified miRNAs, where the backbone is replaced with a pseudopeptide chain. PNAs exhibit remarkable stability against enzymatic degradation and have strong binding capabilities, making them ideal for in vivo applications.

Morpholino Oligomer Modification

We offer morpholino backbone modifications that substitute the ribose sugar with morpholine rings and the phosphate linkage with phosphorodiamidate bonds. This non-ionic structure confers exceptional resistance to nucleases and reduces nonspecific interactions.

Chimeric Antisense Oligonucleotides

By designing chimeric miRNAs that combine different backbone chemistries, we tailor the properties of the molecules to achieve an optimal balance between stability, potency, and specificity. This approach allows for fine-tuning of the therapeutic profile according to client requirements.

Backbone Cyclization

Our team employs cyclization techniques to connect the 3' and 5' ends of miRNAs, forming circular RNAs. This modification enhances nuclease resistance and improves pharmacokinetic properties while maintaining the biological activity of the miRNA.

Key Technologies Included in Our Services

Custom Synthesis Platforms: We utilize advanced solid-phase synthesis techniques for precise incorporation of modified nucleotides.
High-Throughput Screening: Our screening platforms assess the efficacy and specificity of modified miRNAs against target genes.
Analytical Characterization: We perform comprehensive analyses using mass spectrometry, NMR spectroscopy, and HPLC to ensure the quality and integrity of modified miRNAs.
In Vitro and In Vivo Evaluation: Our facilities support functional assays and animal studies to evaluate the therapeutic potential of modified miRNAs.
Regulatory Compliance Support: We provide documentation and guidance to facilitate regulatory submissions, ensuring a smooth transition from research to preclinical development.

Frequently Asked Questions

Q1. What advantages do miRNA backbone modifications offer over unmodified miRNAs?

Backbone modifications enhance the stability of miRNAs against nucleases, improve their pharmacokinetic profiles, and increase binding affinity to target mRNAs. These modifications also reduce immunogenicity and off-target effects, resulting in more effective and safer therapeutic agents.

Q2. How do you determine the best modification strategy for a specific miRNA?

We assess the target gene, desired therapeutic outcome, and delivery method to recommend the most suitable modification. Our experts consider factors such as stability requirements, target tissue, and potential immunogenic responses to customize the modification strategy.

Q3. Can modified miRNAs be used with standard delivery systems?

Yes, modified miRNAs are compatible with various delivery platforms, including lipid nanoparticles, viral vectors, and conjugation with targeting ligands. We optimize the miRNA modifications to enhance compatibility and efficiency with the chosen delivery system.

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.