Currently, in vivo editing is being used for limited diseases, including mucopolysaccharidosis type I (ClinicalTrials.gov: NCT02702115), hemophilia B (ClinicalTrials.gov: NCT02695160), and congenital blindness (ClinicalTrials.gov: NCT03872479). For SCD, in vivo editing resulted in 30% γ-...
Even though not much is known about the exact role of FVIII in neoangiogenesis during embryonic development, work on adult mouse models with hemophilia A has revealed the formation of large and irregular vessels, with high concentrations of vascular remodeling markers, such as αSMA, endoglin, ...