Prenatal Therapy for Congenital Diaphragmatic Hernia and Myelomeningocele: Advances in Particle-Based Delivery

Prenatal intervention for congenital diseases could potentially halt or reverse devastating co-morbidities. Particle-based therapy has emerged as safe option for in-utero treatment delivery. Here, we focus on outlining the current state of knowledge regarding the application of particle-based fetal therapy in the management two structural diseases: congenital diaphragmatic hernia (CDH) and myelomeningocele (MMC).

Intravenous or intra-amniotic injection of particles distribute to various tissues, including the lung, liver, and can cover surfaces such as the spine. They can be modified to target specific tissues and can carry various loads, including protein and genetic material.

In CDH, microRNA loaded particles have successfully reversed the CDH phenotype in animal models. In MMC, particle delivery has resulted in tissue growth and coverage of the spinal defect. Further research is needed for clinical translation in larger animal models and to assess long-term functional outcomes following particle-based interventions.