The AAV capsid plays a crucial role in determining the effectiveness of gene therapy. By engineering AAV capsids, researchers can customize the properties of these vectors to address specific requirements, such as enhancing tissue targeting, reducing immunogenicity, or increasing payload capacity.

Real&Best has created AAV Capsid Libraries for serotypes 2, 5, 6, and 9, carefully screening for variants with diverse tissue tropisms such as the CNS, lungs, kidneys, and muscles. This groundbreaking approach holds enormous promise for developing more potent gene therapies, opening the door to revolutionary treatments that have the potential to change the lives of numerous individuals.

Promoters are integral in the regulation of gene expression within specific tissues or cell types, making them a crucial component of precise and safe gene therapies. The incorporation of tissue-specific promoters into rAAV vectors allows for the selective activation of therapeutic genes solely within particular tissues, ensuring a more tailored and effective approach to treatment. This strategic integration has the potential to revolutionize gene therapy by providing a targeted and controlled method of addressing various disorders while minimizing off-target effects.

Real&Best has developed and established a tissue-specific promoter database that comprehensively encompasses primary tissues including the central nervous system (CNS), skeletal/heart muscle, liver, and kidney. This database serves as a valuable resource for researchers and scientists, providing them with the means to develop gene therapies that are both safe and effective, targeting specific tissues and cell types.

In order to ensure the success of therapeutic interventions, codon optimization is of paramount importance. By strategically aligning the coding sequences with the translational machinery of the host, the efficacy of gene expression can be significantly enhanced.

Real&Best has developed a sophisticated algorithm aimed at preserving DNA unmethylation status, thereby preventing gene silencing. This algorithm also serves to stabilize RNA structure, shielding it from degradation and bolstering its functional stability. Furthermore, it enhances translation efficiency by taking into account rare codons.