α-1 antitrypsin (AAT) is the most common serine protease inhibitor in human plasma and has anti-inflammatory and immunomodulatory properties. AAT deficiency is a well-recognized one-gene disease that is associated with liver failure and early-onset emphysema. The disease can be treated either by delivering the AAT proteins to the body, or, even better, by introducing an intact copy of the AAT protein-producing gene into the recipient's cells to achieve long-term and high levels of AAT production.
AAT is a target protein for gene therapy to address pathologies associated with insufficiencies in native AAT activity or production. The challenge for AAT gene therapy is to achieve long-term and high levels of AAT production. B cells may be engineered to durably express proteins for novel cell-based protein replacement therapy for protein deficiencies. To establish genetically modified B cells that can produce functional AAT, Creative Biolabs has developed B cell gene therapy platforms for therapeutic AAT protein delivery. We have access to a range of technologies and an expert team experienced in engineering B cells to achieve gene transfer at efficiency levels.
Engineered AAT protein-secreting B cells can be applied for the treatment of AAT deficiency diseases including:
Creative Biolabs has proudly launched a B Cell engineering platform. To that end, our team of experts has experience and expertise in the purification, expansion, differentiation, and culture of B cells ex vivo. There are various gene-editing tools available for B cell engineering such as CRISPR technology and viral/non-viral vectors. We will discuss with you and help determine the best-suited technology based on your project. The expression of the transgene is validated by RT-qPCR and Sanger sequencing. We also perform other validation assays such as Western blot, ELISA, and immunocytochemistry upon request. In addition, standard QC assays such as sterility tests and mycoplasma detection are performed to ensure that B cell products are not contaminated.
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