Target cell identification of gene therapies for Cystic Fibrosis
Gene therapies offer an opportunity to prevent or cure the genetic disease cystic fibrosis by addressing the root cause of the disease. However, we have little understanding and control over which cell types can be reached by gene therapies. Here, we join forces with ReCode Therapeutics, a world-leading expert in delivery technologies for therapeutic particles, to elucidate which cell types that are accessible for gene therapies and if this is affected by the genetic defect that causes Cystic Fibrosis. The current standard to identify where gene therapies go after administration are animal experiments that take months or years to conduct and analyze, yet have limited resolution (organ level) and additional risks and costs if findings fail to translate to humans. In this study, we will expose human disease-relevant organ models to gene therapy particles from ReCode Therapeutics. We will establish an assay so that we can rapidly identify at single cell resolution what the target cells are that successfully take up the gene therapy. As such, we will be able to determine if gene therapies are actually delivered to disease-relevant cell types and at a fraction of the costs and time as compared to animal experiments. With genetically engineered cultures that model cystic fibrosis, we will also determine how delivery is affected by the disease. These studies will help overcome the delivery bottleneck and accelerate the implementation of gene therapies for cystic fibrosis and also other genetic diseases.
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