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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.

siRNA Sugar Modification

At Protheragen BioNucleics, we are dedicated to revolutionizing the field of oligonucleotide therapeutics. Leveraging our extensive expertise in Oligonucleotide Drugs CRO, Oligonucleotide Modification Service, and specifically siRNA Modification Service, we are at the forefront of enhancing small interfering RNA (siRNA) technologies. Our focus on sugar modification of siRNA molecules positions us to overcome key challenges in RNA interference therapies, paving the way for more effective and safe gene-silencing solutions.

Understanding siRNA Sugar Modification

siRNA molecules play a crucial role in gene-silencing therapies by guiding the RNA-induced silencing complex (RISC) to degrade target mRNA. However, native siRNA faces significant hurdles, including susceptibility to enzymatic degradation, poor cellular uptake, and potential immunogenicity. Sugar modification involves chemically altering the ribose sugar component of the RNA backbone to improve siRNA properties without compromising its functionality.

By introducing modifications such as 2'-O-methylation, 2'-fluorination, and locked nucleic acids (LNAs), we enhance nuclease resistance, increase binding affinity, and reduce immunogenicity. These modifications are essential for developing siRNA therapeutics with improved stability, specificity, and efficacy, ultimately leading to better outcomes.

Our Comprehensive siRNA Sugar Modification Services

At Protheragen BioNucleics, we offer an extensive suite of siRNA sugar modification services, meticulously designed to meet the unique requirements of your research and development projects.

Custom Synthesis of Modified siRNA

We specialize in synthesizing siRNA with various sugar modifications, providing tailor-made solutions for your specific needs:

2'-O-Methyl (2'-OMe) Modifications: By incorporating 2'-OMe groups, we enhance siRNA stability against nucleases and decrease immune activation, leading to increased half-life in biological systems.
2'-Fluoro (2'-F) Modifications: Our 2'-F modified siRNA exhibits improved binding affinity to target mRNA, resulting in enhanced gene-silencing potency.
Locked Nucleic Acids (LNA): Incorporating LNA nucleotides significantly increases thermal stability and specificity of siRNA duplexes, providing superior performance in gene silencing.

Strategic Design and Optimization

Our scientists work closely with you to design siRNA molecules with optimal modification patterns:

Position-Specific Modifications: We strategically place modified nucleotides to maximize gene silencing while minimizing off-target effects.
Duplex Stability Enhancement: Modifications enhance the thermodynamic properties of siRNA, ensuring efficient incorporation into the RISC complex.
Immune Response Mitigation: Designing siRNA to reduce activation of the innate immune system, improving safety profiles.

Advanced Analytical and Characterization Services

We offer comprehensive analytical services to characterize modified siRNA:

Mass Spectrometry Analysis: Confirming the molecular weight and integrity of modified siRNA.
High-Performance Liquid Chromatography (HPLC): Ensuring high purity and quality of synthesized siRNA.
Melting Temperature Determination: Assessing duplex stability through thermal analysis.

In Vitro and In Vivo Functional Validation

To guarantee the efficacy of modified siRNA, we provide robust validation services:

Cellular Uptake and Localization Studies: Using fluorescence labeling to monitor siRNA internalization and distribution within cells.
Gene Knockdown Efficiency Testing: Quantitative PCR and Western blot analyses to measure target gene expression levels post-treatment.
Animal Studies: Pharmacodynamic and pharmacokinetic assessments in relevant animal models to evaluate therapeutic potential and biodistribution.

Regulatory Compliance and Documentation Support

Understanding the complexities of regulatory pathways, we assist in compiling comprehensive documentation. We provide detailed reports and data packages required for investigational new drug (IND) applications.

Frequently Asked Questions

Q1: What advantages do sugar modifications offer over other siRNA modifications?

Sugar modifications specifically enhance nuclease resistance and binding affinity without significantly altering the siRNA's ability to engage the RISC complex. This approach improves the stability and efficacy of siRNA therapeutics while minimizing potential side effects associated with other modifications.

Q2: How do you ensure the specificity of modified siRNA to the target gene?

We employ advanced computational tools and empirical validation to design siRNA sequences with high specificity. Strategic placement of sugar modifications reduces off-target interactions, ensuring precise gene silencing.

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.