tRNA Therapeutics Development
The field of tRNA therapeutics has witnessed remarkable advancements in recent years, offering promising avenues for the therapeutics of various diseases. Our company's commitment to rare disease research and development positions us as a leader in advancing tRNA therapeutics for these challenging conditions. Our expertise in tRNA biology, RNA-based therapeutics, and gene therapy enables us to provide tailored solutions to our clients' needs.
Overview of tRNA Therapeutics
Transfer RNA (tRNA) is a fundamental component of the protein synthesis machinery within cells. Traditionally, tRNA has been primarily recognized for its role in carrying amino acids to the ribosomes during translation. However, recent research has shed light on the untapped potential of tRNA beyond its canonical function. tRNA can be enzymatically cleaved, giving rise to small RNA fragments known as tRNA-derived small RNAs (tsRNAs) or tRNA fragments (tRFs).
Fig.1 Basic structure and function of tRNA. (Suzuki, Tsutomu, 2021)
Two types of single-gene diseases may be solved by tRNA gene therapy, namely diseases related to nonsense mutations and AARS mutation diseases related to tRNA depletion. tRNA has emerged as a key player in gene regulation, with the ability to modulate gene expression at both transcriptional and translational levels. These RNA molecules possess unique features that make them attractive candidates for therapeutic interventions, offering a new dimension in the field of gene therapy.
tRNA Therapeutics for Rare Diseases
Rare diseases often result from mutations in specific genes, leading to impaired protein function or expression. tRNA therapeutics offer a promising avenue for treating rare diseases by precisely targeting the underlying genetic defects. By restoring normal gene expression patterns or compensating for dysfunctional proteins, tRNA-based interventions hold the potential to provide transformative therapies for rare diseases.
Table 1 Rare diseases caused by aberrant tRNA modifications. (Suzuki, Tsutomu, 2021)
| Rare Disease | Gene | RNA Modification | tRNA Species |
|---|---|---|---|
| MELAS | mt-tRNALeu(UUR) gene | τm5U | mt-tRNA |
| MERRF | mt-tRNALys gene | τm5s2U | mt-tRNA |
| MLASA | PUS1 | Ψ | cyto-tRNA, mt-tRNA |
| Familial dysautonomia | ELP1 | mcm5U and derivatives | cyto-tRNA |
| Amyotrophic lateral sclerosis | ELP3 | mcm5U and derivatives | cyto-tRNA |
| HSD10 disease | SDR5C1 | m1A, m1G | mt-tRNA |
Our Services
Our company collaborates with pharmaceutical and biotechnology partners to design and optimize tRNA therapeutics for various rare disease indications. Leveraging our deep understanding of tRNA biology and gene regulation, we employ state-of-the-art computational tools and experimental approaches to design tRNA therapeutics with enhanced efficacy and safety profiles. We are at the forefront of the development of tRNA therapeutics for rare diseases and provide cutting-edge rare disease therapeutic solutions and services to accelerate the transformation of this breakthrough technology.
tRNA Therapeutics Design
- Sequence identification
- Machine learning-guided sequence and modification optimization
- High throughput quantification and characterization using tRNA functional assays
Optional tRNA Therapeutics
- Providing suppressor-tRNA (sup-tRNA) to introduce the correct amino acids to suppress premature termination codon (PTC) associated with nonsense mutations.
- Exogenous tRNA are supplemented as a compensatory measure to mitigate the impact of missense mutations in proteins essential for tRNA biosynthesis and translation functionality.
tRNA Therapeutics Delivery
Why Choose Us?
With many years of experience and a proven track record of quality, innovation, and customer support, our company provides comprehensive services for leading and emerging biopharmaceutical companies and scientific research institutions to develop tRNA therapeutics for rare diseases. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
Reference
- Suzuki, Tsutomu. "The expanding world of tRNA modifications and their disease relevance." Nature Reviews Molecular Cell Biology 22.6 (2021): 375-392.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.