Loss of glycosyltransferase function during cellular glycosylation will cause overall changes in the glycan structure produced, leading to corresponding diseases. Precise, stable, and systematic gene editing technology targets specific sites in the cell genome to re-edit the cell's glycosylation and is used to treat single-gene diseases or complex diseases with relevant specific gene targets. Zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and clustered regularly interspaced palindromic repeats (CRISPR)/Cas9 can achieve stable targeted knockout, knock-in, and regulation of glycogene expression.
Fig.1 Overview of therapeutic genome editing strategies. (van Haasteren, et al., 2020)
While some complex or idiopathic diseases are treated by targeting specific genes, the key to success depends on how efficiently the editing machinery is delivered to target cells and tissues in vivo or in vitro. CD BioGlyco has developed Glycogene Delivery Services to effectively deliver editing components such as deoxyribonucleic acid (DNA), ribonucleic acid (RNA), or proteins to targets through viral vectors, physical or chemical methods.
Liposomes are widely used in many clinical fields such as vaccine and gene-drug delivery, cancer treatment, and tumor imaging. Liposome-mediated gene transfection is one of the earliest and most widely used non-viral transfection strategies for gene therapy. In gene delivery research, the cationic lipids we use include but are not limited to:
Fig.2 Lipid transfection-based delivery services. (CD BioGlyco)
Liposomes are not only used as DNA vaccine delivery carriers but are also widely used for the delivery of various types of RNA. For example, lipid-based or liposome-modified small interfering RNA (siRNA) has been widely used in the treatment of eye diseases and tumors. Compared with siRNA, short hairpin RNA (shRNA) has higher potency and sustainable effects for targeted delivery of Toll-like receptor 4 (TLR4).
CD BioGlyco has developed a Glycogenomic Platform to provide Glycogene Editing, delivery, and Expression Analysis services to our clients worldwide. If you are interested in our services, please feel free to contact us for more information.