A road less travelled: large animal models in immunological research

WR Hein, PJ Griebel - Nature Reviews Immunology, 2003 - nature.com
The main advances in immunology have been forged or underpinned by animal
experiments. However, animal research now focuses excessively on one laboratory species …

In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application

G Almeida-Porada, A Atala, CD Porada - Molecular Therapy-Methods & …, 2016 - cell.com
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 …

Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes

TC MacKenzie, GP Kobinger, NA Kootstra, A Radu… - Molecular Therapy, 2002 - cell.com
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 …

Prenatal somatic cell gene therapies: charting a path toward clinical applications (proceedings of the CERSI‐FDA meeting)

A Herzeg, G Almeida‐Porada, RA Charo… - The Journal of …, 2022 - Wiley Online Library
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 …

In utero stem cell transplantation and gene therapy: Recent progress and the potential for clinical application

LE McClain, AW Flake - Best practice & research Clinical obstetrics & …, 2016 - Elsevier
Advances in prenatal diagnosis have led to the prenatal management and treatment of a
variety of congenital diseases. Although surgical treatment has been successfully applied to …

Gene therapy for the fetus: is there a future?

AL David, D Peebles - Best Practice & Research Clinical Obstetrics & …, 2008 - Elsevier
Gene therapy uses the intracellular delivery of genetic material for the treatment of disease.
A wide range of diseases–including cancer, vascular and neurodegenerative disorders and …

Induction of stable prenatal tolerance to β-galactosidase by in utero gene transfer into preimmune sheep fetuses

ND Tran, CD Porada, G Almeida-Porada… - Blood, The Journal …, 2001 - ashpublications.org
The successful transduction of hematopoietic stem cells and long-term (28 months)
transgene expression within the hematopoietic system following the direct injection of high …

Physiological parameter values for physiologically based pharmacokinetic models in food‐producing animals. Part III: Sheep and goat

M Li, YS Wang, T Elwell‐Cuddy… - Journal of veterinary …, 2021 - Wiley Online Library
This report is the third in a series of studies that aimed to compile physiological parameters
related to develop physiologically based pharmacokinetic (PBPK) models for drugs and …

In utero gene therapy: current challenges and perspectives

SN Waddington, MG Kramer, R Hernandez-Alcoceba… - Molecular Therapy, 2005 - cell.com
Over the past few years, considerable progress in prenatal diagnosis and surgery combined
with improvements in vector design vindicate a reappraisal of the feasibility of in utero gene …

Rhesus monkey model for fetal gene transfer: studies with retroviral-based vector systems

AF Tarantal, JP O'rourke, SS Case, GC Newbound… - Molecular Therapy, 2001 - cell.com
Many life-threatening conditions that can be diagnosed early in gestation may be treatable
in utero using gene therapy. In order to determine in utero gene transfer efficiency and …