Development of AAV Vector for Rheumatoid Arthritis Research
Rheumatoid arthritis (RA) is a systemic disease characterized by chronic inflammation and synovial hyperplasia leading to the destruction of cartilage and bone. During the past, novel therapeutic biologic agents, such as TNF-α and IL-1 inhibitors, have been developed. However, their side effects, short duration of effect, need for long-term treatment make them not optimal RA therapy. In this condition, the development of targeted gene therapy is an increasingly attractive option for long-term RA therapy. As a pioneer company in gene therapy for years, Creative Biolabs provides AAV vectors development service for RA research.
Gene Therapy for RA
Gene therapy is a novel promising approach for treating diseases by delivering therapeutic genes to specific organs or tissues. Of the viral vectors that have been used to deliver the gene of interest, recombinant AAV vectors are particularly useful in targeting slowly dividing cells and in the treatment of chronic disease because of their potential for site-specific integration into the host genome or formation of stable episomal DNA, both of which result in long term gene expression. AAV2 has been the most widely studied serotype in clinical trials in hemophilia B, leucodystrophy, cystic fibrosis, RA and other conditions. To date, no adverse events due to the vector have been observed and long-term protein expression has been achieved. What's more, most cells found in the joint can be transduced in vitro by rAAV2, including human chondrocytes, human and murine FLS, human macrophages.
Figure 1. Local in vivo gene therapy for RA. (Adriaansen, 2006)
Candidate Targets for RA Gene Therapy
Currently, several therapeutic targets for gene therapy have been developed, including but not limited to pro-inflammatory cytokine, anti-inflammatory cytokines, and transcription factor NF-κB. In humans, biologicals targeting cytokines and their receptors have been proven useful as specific therapies for RA. Anti-inflammatory cytokines are also potential targets because they can suppress and counterbalance Th1-driven responses and inhibit the production of pro-inflammatory cytokines. What's more, transcription factor NF-κB may also be a good target for gene therapy. These molecules may be used simultaneously, either by gene therapy or by combining gene therapy with systemic biological therapy.
Services
With years of experience, Creative Biolabs has developed an advanced technology platform for AAV vector design and construction. To avoid the potential immunogenicity of the AAV vector, our professional scientists will help global customers switch serotype or modify capsid to construct high-efficacy recombinant AAV vectors. Furthermore, we also offer pharmacokinetics, biodistribution and toxicity evaluation services to ensure the safety of vectors used for gene therapy.
If you are interested in the service we offer, please feel free to contact us for more information.
Reference
- Adriaansen, J.; et al. (2006). Gene therapy as a therapeutic approach for the treatment of rheumatoid arthritis: innovative vectors and therapeutic genes. Rheumatology. 45(6), pp.656-668.
