Objective: Prenatal ex vivo gene therapy might be an effective and safe strategy with which
to treat severe genetic disorders in utero. For this purpose, autologous fetal stem cells must …

Prenatal gene therapy aims to deliver genes to cells and tissues early in prenatal life,
allowing correction of a genetic defect, before long‐term tissue damage has occurred. In …

Introduction: Fetal gene therapy (FGT) can potentially be applied to perinatally lethal
monogenic diseases for rescuing clinically severe phenotypes, increasing the probability of …

Following fetal or neonatal gene transfer in mice and other species immune tolerance of the
transgenic protein is frequently observed; however the underlying mechanisms remain …

OBJECTIVE: To determine if mesenchymal stem cells (MSC) derived from human fetal
pancreatic tissue (pMSC) would engraft and differentiate in sheep pancreas following …

The intracelomic route for in utero hematopoietic stem cell transplantation was evaluated in
preimmune fetal sheep and the engraftment characteristics were defined. Twelve twin ovine …

Adenovirus and adeno-associated virus vector-mediated gene delivery is limited by the
induction of a humoral immune response that prevents readministration. To determine …

Gene editing for the cure of inborn errors of metabolism (IEMs) has been limited by
inefficiency of adult hepatocyte targeting. Here, we demonstrate that in utero CRISPR/Cas9 …

Objective: The biologic explanation for fetal receptivity to donor engraftment and subsequent
long-term tolerance following transplantation early in gestation is not known. We …

Hemophilia A represents the most common inheritable deficiency of the coagulation
proteins. Current state-of-the-art treatment consists of frequent prophylactic infusions of …