vectors & gene therapy distribution

As biotherapy experts, we specialize in providing a range of assays to meticulously evaluate the biodistribution of gene therapy vectors, including AAV, and the effectiveness of gene therapy.

Assessing the biodistribution of viral vectors in gene therapy research traditionally relies on reporter gene expression, using fluorescent or luminescent signals to identify transduced tissues and cells. However, AAV vectors have limited DNA capacity, hindering the co-expression of therapeutic and reporter genes. Additionally, immune responses to fluorescent proteins like GFP can cause damage, especially in the central nervous system. Relying solely on reporter gene expression assumes equivalent transduction efficiency, which may not be accurate. To address these issues, it is essential to evaluate viral vector biodistribution directly, ensuring a more comprehensive understanding of their in vivo distribution.

Cilcare’s capabilities include, but are not limited to:

• Tissue processing: collection, fixation, embedding and sectioning
• H&E, immunohistochemistry, immunofluorescence
• Microscopy
• qPCR: quantification of the presence of the therapeutic gene in different tissues or organs