Gene Therapy Vectors
Examining the safety and biodistribution of different vectors for gene therapy delivery in the prenatal period.
Successful, safe gene therapy technologies often need equally safe and efficacious delivery vector to reach their intended target cells. Our lab focuses on technologies like lipid nanoparticles (LNPs) and adeno-associated viruses (AAV) to ascertain parameters such as biodistribution, minimum effective dose, immunogenicity and toxicity.