Why Nucleic Acid-based Therapy is Great for Ocular Diseases

Nucleic acid-based therapy is an innovative form of drug delivery that can be delivered to cells in vitro or in vivo using viral or nonviral vectors. Recent technological advances have demonstrated the molecular basis of various eye diseases, and the eye diseases most likely to benefit from nucleic acid-based therapy include genetic disorders such as retinitis pigmentosa, retinoblastoma, or melanoma, among other tumors, and acquired proliferative and neovascular retinal diseases. This is due to the immune privilege of the human eye and the diverse selection of ocular genes (researchers have isolated 55 genes from the eye and mapped 118 retinal disease loci) making them well suited to successful nucleic acid-based therapy, among other things. Additionally, ocular disorders allow for the topical application of therapeutic agents, reducing the risk of systemic effects. Therefore, in addition to being a method for studying disease pathogenesis, nucleic acid-based therapy is also a very promising therapeutic modality for ocular diseases.

Fig. 1. An overview of retinal gene therapy strategies. (Buck TM, et al., 2020)

Service Overview

Ace Therapeutics has been committed to developing novel treatments for ocular diseases for decades. We are proud of our extensive and growing contribution to nucleic acid-based therapy for ocular diseases. With our talented scientists' comprehensive knowledge of ophthalmology and genetics, we provide you with a full science pathway to support your program from start to finish.

Ace Therapeutics has a fully professional research team responsible for the entire development of your project, this includes the production of plasmids and vectors, to the generation of custom models for the corresponding ocular disease. Whether you are developing viral vector-based therapeutics such as adeno-associated virus (AAV) or lentiviral (lenti) products, or applying gene editing technologies such as CRISPR/cas9, you will benefit from in-depth research knowledge and experience. Beyond that, our scientists can work with your in-house teams to secure key development milestones and support you with critical decisions along the way.

According to the project research needs of global customers, Ace Therapeutics provides the following related services for development of nucleic acid-based therapy for ocular disease, including but not limited to.

Our Areas of Expertise

• Retinal disease.
• Age-related macular degeneration.
• Hereditary eye disease.
• Glaucoma.
• Corneal disease.
• Optic nerve disease.

Nucleic acid-based therapy success depends on creating safe and efficient gene delivery vectors. Ace Therapeutics' talented scientists design and construct viral and non-viral vectors for ophthalmic nucleic acid-based therapy development. In addition, we provide gene editing services to advance basic nucleic acid-based therapy research. If you are interested in our services or need more detailed information, please feel free to contact us. Our experienced scientists are ready to help you!

References

1. Buck TM, Wijnholds J. Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays. Int J Mol Sci. 2020 12;21(12):4197.
2. Ghoraba HH, Akhavanrezayat A, Karaca I, et al. Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses. Clin Ophthalmol. 2022 Jun 3;16:1753-1771.

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