Therapeutic
Oligos in the Therapeutic Applications
Since oligonucleotides can be customized to specific DNA or RNA sequences, their precise design allows them to bind to target nucleic acids, thereby modulating gene expression or modifying genetic information. This specificity and versatility make oligonucleotides valuable tools in both research and therapeutic applications. Eurofins Genomics offers a variety of oligonucleotides for the therapeutic industry, including:
- sgRNA, Antisense oligo ASO, siRNA
- Indexing Oligos
- Barcoded adapters
- Enrichment panels
- Library prep oligos
Application Areas for Oligos in Therapeutics
Antisense Oligonucleotides (ASOs)
Antisense oligonucleotides are designed to bind to complementary mRNA sequences, blocking the translation of target genes. This mechanism can downregulate the expression of disease-associated genes. ASOs have shown promise in treating a variety of conditions, including genetic disorders, cancers, and viral infections. Notable examples include:
- Nusinersen: Approved for the treatment of spinal muscular atrophy (SMA), this ASO increases the production of the survival motor neuron (SMN) protein by modifying splicing of the SMN2 gene.
- Mipomersen: Used to lower cholesterol levels in patients with familial hypercholesterolemia by inhibiting the synthesis of apolipoprotein B-100.
Order RNA
More on ASO oligos
Small Interfering RNAs (siRNAs)
siRNAs are double-stranded oligonucleotides that trigger the RNA interference (RNAi) pathway, leading to the degradation of specific mRNA molecules. This results in the silencing of gene expression. siRNAs are particularly effective in targeting diseases at the molecular level. Examples include:
- Patisiran: The first FDA-approved siRNA therapeutic, used to treat hereditary transthyretin-mediated amyloidosis by reducing the production of the transthyretin protein.
Aptamers
Aptamers are single-stranded oligonucleotides that fold into unique three-dimensional structures, allowing them to bind to specific proteins, peptides, or small molecules with high affinity and specificity. They can act as antagonists, inhibiting the function of their targets, or as delivery agents. Key examples are:
- Pegaptanib: An aptamer used to treat age-related macular degeneration by inhibiting vascular endothelial growth factor (VEGF).
Gene Editing
Oligonucleotides play a critical role in gene editing technologies such as CRISPR-Cas9. In this context, guide RNAs (gRNAs) direct the Cas9 enzyme to specific genomic locations, enabling precise gene modifications. This has transformative potential for treating genetic diseases by correcting mutations at their source.
Advancements in Delivery Systems
Effective delivery of oligonucleotide therapeutics remains a significant challenge. Innovative delivery systems are being developed to enhance stability, target specificity, and cellular uptake of oligonucleotides. These include:
- Lipid Nanoparticles (LNPs): Widely used for the delivery of siRNAs, such as in the case of patisiran.
- GalNAc Conjugates: Leveraged for targeted delivery to hepatocytes, enhancing the therapeutic index of oligonucleotides like ASOs and siRNAs.
Regulatory and Commercial Landscape
The therapeutic potential of oligonucleotides has led to increased regulatory approvals and commercial interest. The FDA and EMA have established frameworks for the evaluation and approval of oligonucleotide therapeutics, fostering a growing market. Numerous oligonucleotide-based drugs have received approval, and many more are in clinical trials, reflecting the expanding role of these molecules in modern medicine.
Conclusion
Oligonucleotides represent a rapidly evolving class of therapeutics with the potential to address a wide array of medical conditions. Their ability to precisely target genetic material offers unparalleled opportunities for the treatment of diseases at the molecular level. As research progresses and delivery technologies improve, oligonucleotide therapeutics are poised to become a cornerstone of personalized medicine, offering hope for patients with previously intractable conditions.