There are promising results in treating genetic disorders found in introducing modified genetic material into tissue or cells, known as gene therapy, and recombinant adeno-associated viruses (AAVS).
Image Credit: ACROBiosystems
AAV capsids act as a guide while moving genetic material into host cells and tissue. Research has shown that AAV serotypes are developed concurrently to identify the best subtype for preclinical and clinical use.
There has been limited success found in clinical practices of gene therapy, potentially due to the variable effectiveness of transgene delivery between in vitro/in vivo models and humans.
This study mimics human physiology in an organoid-based approach with organ-specific features and cell diversity within the organ.
Organoids provide a more in-depth model than traditional 2D cell cultures by modeling cell signaling, cell-extracellular and cell-cell matrix interactions to better simulate the intricate balance between varying cell types and their functions.
This study used RNA sequencing to verify several human-based organoid models by identifying known marker genes. The transgene efficacy was evaluated through five AAV serotypes on each organoid model.
A comparison of the expression of delivered GFP transgene between each organoid was used to assess transduction to select the optimal AAV serotype.
This research shows improved preclinical modeling in using human organoid models when selecting AAV serotypes and evaluating the effectiveness of transgene delivery. This provides better guidance for gene therapy development when advancing into clinical research.
Read the full app note
About ACROBiosystems
ACROBiosystems is a leading manufacturer of recombinant proteins and other critical reagents to support the development of target therapeutics. The company employs an application-oriented development strategy, with a particular focus on product design, quality control, and solution-based support. Our products and services enable anyone in the field of drug development to have a more intuitive and streamlined process.
To respond to the coronavirus pandemic, ACROBiosystems has developed SARS-CoV-2 antigens specifically designed and optimized for serological test kits, including Spike-derived antigen S1, RBD, and Nucleocapsid protein. Proteins have been supplied to diagnostic companies in large quantities.
Sponsored Content Policy: News-Medical.net publishes articles and related content that may be derived from sources where we have existing commercial relationships, provided such content adds value to the core editorial ethos of News-Medical.Net which is to educate and inform site visitors interested in medical research, science, medical devices and treatments.