Abstract Purpose of Review Prenatal stem cell and gene therapy approaches are among the
few therapies that can promise the birth of a healthy infant with specific known genetic …

Abstract Purpose of Review The purpose of this review is to summarize the current status of
in utero gene therapy and postnatal in vivo genome editing with an eye toward prenatal …

In utero stem cell transplantation, which promises treatment for a host of genetic disorders
early in gestation before disease effect stems from Ray Owen's seminal observation that self …

Current concerns over insertional mutagenesis by retroviral vectors mitigate investigations
into alternative, potentially persistent gene therapy vector systems not dependent on …

Objective: Using fetal goats as animal models, to establish the methodology of in utero
transplantation of human hematopoeitic stem cell (HSC) under B-scan ultrasonographic …

Intrauterine gene therapy (IUGT) potentially enables the treatment and possible cure of
monogenic diseases that cause severe fetal damage. The main benefits of this approach will …

Background Prenatal gene transfer may enable early causal intervention for the treatment or
prevention of many devastating diseases. Nevertheless, permanent correction of most …

Application of gene therapy in utero has been considered as a strategy for treatment or even
prevention of early onset genetic disorders such as cystic fibrosis and Duchenne muscular …

Advances in prenatal imaging, molecular diagnostic tools, and genetic screening have
unlocked the possibility to treat congenital diseases in utero prior to the onset of disease …