Ribozymes Therapeutics Development
Selective gene silencing through ribozymes provides researchers with a new strategy for blocking gene expression and validating drug targets. Ribozymes have shown promising progress in the therapeutics of rare diseases. As a company dedicated to the development of rare disease research, our company is dedicated to providing clients with ribozymes therapeutics for rare disease development services.
Introduction to Ribozymes
Ribozymes (or RNA enzymes) are catalytic RNA capable of cleaving target RNA molecules in a sequence-specific manner. They can be utilized to cut target RNA, thereby reducing gene expression. Ribozymes primarily include hammerhead ribonuclease, hairpin ribonuclease, and hepatitis delta ribonuclease. Among these, the hammerhead ribonuclease is the most widely used in the field of gene therapy and has found extensive applications in gene therapy.
Fig.1 Examples of RNA processing mediated by ribozymes. (Lau, M. W. L., and A. R. Ferré-D, 2016)
The characteristics required for therapeutic nucleic enzymes include:
- Good catalytic activity under physiological conditions
- Resistance to nucleases
- Small size
- Easy to synthesize
- Non-restrictive substrate recognition rules
Ribozymes Therapeutics for Rare Diseases
Ribozymes show tremendous promise in the therapeutics of rare diseases, and their development as therapeutics has made significant progress. The ability of nucleic acid enzymes to selectively degrade pathogenic RNA can be achieved through one of two possible methods. The first is a vector-based method involving intracellular expression of nuclease-encoding vectors. The second method utilizes chemically synthesized nucleic enzymes for exogenous delivery, where both large nucleic enzymes and small nucleic enzymes are suitable for carrier-based delivery.
Table 1 Rare disease research with ribozymes therapeutics. (Wong, Bao Chi, et al., 2022)
| Title | Ribozyme | Target gene | Disease | NCT number | Time |
|---|---|---|---|---|---|
| Gene Therapy in HIV-Positive Cases with Non-Hodgkin's Lymphoma | L-TR / Tat-neo | Tat, Rev mRNA | Non-Hodgkin lymphoma, HIV infections | NCT00002221 | 2001 – N/A |
Our Services
With many years of rich experience, our company comprehensively considers the plasticity of ribozymes, the selection of optimal target sites, cleavage efficiency, transfection efficiency, stability within recipient cells, and other factors, to provide you with quality ribozymes therapeutics development services to help you more efficiently carry out the research and development of rare disease therapy. Our services include but are not limited to:
Ribozyme Screening
- Selection of the target gene of ribozyme
- Selection of the optimal design of the ribozyme
- Selection of the oligomer's nucleotide backbone
Sequences and Structural Predictions
- Sequence alignment
- Secondary structure prediction
- Motif prediction
Ribozymes Delivery
- Viral vector delivery
-AAV, lentivirus, retrovirus. - Nonviral vector delivery
-Electroporation, microinjection, lipid delivery
Project Workflow
As a biotechnology company with substantial expertise in ribozyme therapeutics development, our company is committed to providing our clients with effective, customized rare disease approaches through research. If you are interested in our ribozymes therapeutics development services, please feel free to contact us for more details and quotation information of related services.
References
- Lau, M. W. L., and A. R. Ferré-D. "Riboswitches and Ribozymes." (2016): 465-473.
- Wong, Bao Chi, et al." Ribozymes as therapeutic agents against infectious diseases." RNA Therapeutics-History, Design, Manufacturing, and Applications. In-Tech, 2022.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.