Solutions for gene therapy using a deeper analytical characterization of AAV vector development

Gene therapy—a type of DNA-based medicine that utilizes a virus to insert a healthy gene into cells to replace a mutated, disease-causing variant has emerged as one of the most promising medical treatments. While great advances and successfully launched treatments are available, there is still a lot of work that needs to be done to better characterize and further optimize viral vectors.

We share examples of using Prometheus to precisely monitor and distinguish unfolding and aggregation behavior of different adeno-associated virus (AAV) serotypes. We will show how concentration, buffer formulation and various stresses influence the stability of viral particles. Together, these results enable scientists to screen, identify, characterize and select the top AAV candidates that can be developed into novel gene therapies with the greatest potency and highest efficacy.

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Practical strategies for overcoming challenges in the development of AAV vectors for gene therapy
Practical strategies for overcoming challenges in the development of AAV vectors for gene therapy

Gene therapy promises to treat and potentially cure a disease by correcting its underlying genetic cause. W...

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Francisella novicida Cas9 interrogates genomic DNA with very high specificity and can be used for mammalian genome editing
Francisella novicida Cas9 interrogates genomic DNA with very high specificity and can be used for mammalian genome editing

Trying to develop safe and effective gene therapies?

Biophysical methods can help.

Find out how