Clinical trials in cystic fibrosis (CF) patients established proof-of-principle for transfer of the
wild-type cystic fibrosis transmembrane conductance regulator (CFTR) gene to airway …

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 clinical …

Gene replacement therapy is an attractive approach for treatment of genetic disease, but
may be complicated by the risk of a neutralizing immune response to the therapeutic gene …

Recent advances in high-throughput molecular testing have made it possible to diagnose
most genetic disorders relatively early in gestation with minimal risk to the fetus. These …

The major complication associated with protein replacement therapy currently used in the
treatment of hemophilia B (HB) is the development of antibodies to the infused human Factor …

In this study we investigate the efficacy of lentiviral vectors of different pseudotypes for gene
transfer to tissues of the preimmune fetus. BALB/c fetuses at 14–15 days' gestation received …

We are living in a golden age of medicine in which the availability of prenatal diagnosis, fetal
therapy, and gene therapy/editing make it theoretically possible to repair almost any defect …

Increasing use of hematopoietic stem cells for retroviral vector–mediated gene therapy and
recent reports on insertional mutagenesis in mice and humans have created intense interest …

Correction of perinatally lethal neurogenetic diseases requires efficient transduction of
several cell types within the relatively inaccessible CNS. Intravenous AAV9 delivery in …

Introduction Inborn errors of metabolism (IEMs) often result from single-gene mutations and
collectively cause liver dysfunction in neonates leading to chronic liver and systemic …