Gene therapy

Overview of Gene Therapy

Gene therapy is a medical strategy focused on inducing therapeutic outcomes by modulating gene expression or modifying the biological characteristics of living cells. It encompasses a range of techniques, such as introducing or manipulating genes, somatic and germline gene therapy, in vivo versus ex vivo therapies, and gene editing. Currently, numerous gene therapy treatments have received approval. These strategies typically employ adeno-associated viruses (AAVs) and lentiviruses for gene integration, performed in vivo and ex vivo, respectively. Delivery vectors include recombinant viruses as well as naked DNA or DNA complexes.

Bispecific Antibodies (BsAbs) in Gene Therapy

BsAbs serve as valuable tools for guiding gene transfer vectors to target cells. Their design flexibility allows simultaneous binding to viral surface proteins and cell surface molecules, acting as adapters to link vectors to specific cell markers. This avoids structural modifications to the virus, preserving vector assembly, activity, and stability. Once an effective adapter is established, derivatives of the vector can deliver various therapeutic genes. Additionally, BsAbs themselves can function as therapeutic genes in gene therapy. Compared to protein injections, local expression of BsAbs via gene therapy offers sustained and effective antibody levels, improved biodistribution, and enhanced tumor penetration, potentially overcoming clearance issues.

Creative Biolabs offers a diversity of selections of over a hundred BsAb options to aid in gene therapy research. Customization options are accessible to adapt BsAbs for gene therapy requirements, catering to specific research demands.

Fig. 1 Delivery of BsAbs. ¹