lncRNA Therapeutics Development
Long non-coding RNA (lncRNA) is a type of regulatory RNA molecule. Our company has long-term devoted to the development and application of RNA-based therapy. With years of experience, our scientists have developed a lncRNA therapeutics development platform to boost our global customers' research and project goals. We are pleased to use our rich experience and advanced platform to provide the best services to meet our customers' demand in rare disease research and development.
Introduction to lncRNAs
Long non-coding RNAs (lncRNAs) are RNAs that are greater than 200 nucleotides in length and cannot encode proteins. According to the relative positions of lncRNA coding sequences and protein-coding genes, they can be divided into sense lncRNA, antisense lncRNA, bidirectional lncRNA, intronic lncRNA, and intergenic lncRNA. Non-coding RNA is involved in a variety of complex biological processes, such as dosage compensation effect, epigenetic regulation, cell cycle regulation, cell differentiation regulation, and many other life activities. Therefore, targeted therapy of non-coding RNAs can be used to treat various diseases.
Fig.1 Significance of miRNA Therapeutics. (Chakraborty, Chiranjib, et al., 2021)
lncRNA Therapeutics for Rare Diseases
lncRNAs exhibit cell-, tissue-, and tumor-specific expression, making them excellent candidates for rare diseases. Combined with high specificity and the accelerated development of nucleic acid therapies, lncRNA may become an easily accessible therapeutic target, and lncRNA therapeutics will play an important role in the research and development of rare disease therapies. There have been some cases of lncRNA therapeutics being used in rare disease research:
Table 1 Long non-coding RNAs and human rare diseases. (He, J. H., et al., 2014)
| Name | Location | Length (bp) | Dysfunction type | Associated diseases |
|---|---|---|---|---|
| C15orf2 | Chr15: 24920541–24928593 | 8053 | Expression | Prader-Willi syndrome |
| H19 | Chr11: 2016406–2019065 | 2559 | Epigenetics | Beckwith-Wiedemann |
| Ube3a-as | Unknown | Unknown | Locus | Angelman syndrome |
| KCNQ1OT1 | Chr11: 2661768–2721228 | 91671 | Epigenetics | Beckwith-Wiedemann syndrome |
| DGCR5 | Chr12: 18958027–18982141 | 3334 | Expression and mutation | DiGeorge syndrome |
| NRON | Chr9q33.3: 129270966–129481601 | 2730 | Expression | Down's syndrome |
| XIST | ChrX: 73040495–73072588 | 31093 | Epigenetics | Klinefelter's syndrome |
| Ak042766 | Unknown | 1029 | Expression | Restless legs syndrome |
| BX118339 | Chr6: 214865 45–21512123 | 25578 | Mutation | West syndrome |
| ASFMR1 | ChrX: 146990949–147003676 | 3026 | Expression | Fragile X syndrome |
| CECR3 | Chr22: 17737750–17747623 | 1915 | Expression | Cat eye syndrome |
| CECR9 | Chr22: 17809924–17810122 | 198 | Expression | Cat eye syndrome |
| BPESC1 | Chr3: 138823027–138844009 | 3518 | Mutation | Blepharophimosis syndrome |
Our Services
With an advanced lncRNA therapeutics development platform, our scientists are adept at using long non-coding RNA molecules to regulate gene expression to treat rare diseases and have extensive experience in cooperation with global pharmaceutical companies. We provide our clients with tools and methods to study lncRNA therapeutics and design tissue- and cell-type-specific genomic drugs.
lncRNA Therapeutics Design
Multivalent synthetic long non-coding RNA (lncRNA) therapeutics are formed by assembling functional lncRNA structures on a minimal chassis.
lncRNA Therapeutics Modification
Chemical modifications to the ribose moiety, nucleotide, or phosphorothioate backbone increase stability and prevent nucleases.
lncRNA Therapeutics Delivery
Taking into account the higher molecular weight and conserved secondary structure of lncRNA, we provide viral vector and non-viral vector delivery strategies to provide you with customized solutions.
Project Workflow
With the participation of multidisciplinary researchers with extensive project experience, our company is committed to providing one-stop development services to help you accelerate lncRNA therapeutics development for rare diseases. If you are interested in our lncRNA therapeutics development services, please feel free to contact us for more details and quotation information on related services.
References
- Liu, Ying, et al. "Long non-coding RNAs: Biogenesis, functions, and clinical significance in gastric cancer." Molecular Therapy-Oncolytics 23 (2021): 458-476.
- He, J. H., et al. "Association between long non‑coding RNA and human rare diseases." Biomedical reports 2.1 (2014): 19-23.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.