Center for fetal monkey gene transfer for heart, lung, and blood diseases: an NHLBI resource for the gene therapy community

Abstract

The goals of the National Heart, Lung, and Blood Institute (NHLBI) Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases are to conduct gene transfer studies in monkeys to evaluate safety and efficiency; and to provide NHLBI-supported investigators with expertise, resources, and services to actively pursue gene transfer approaches in monkeys in their research programs. NHLBI-supported projects span investigators throughout the United States and have addressed novel approaches to gene delivery; "proof-of-principle"; assessed whether findings in small-animal models could be demonstrated in a primate species; or were conducted to enable new grant or IND submissions. The Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases successfully aids the gene therapy community in addressing regulatory barriers, and serves as an effective vehicle for advancing the field.

FIG. 1.

Annual call for Letters of Intent.

FIG. 2.

Examples of a subset of animals transferred prenatally during the late first trimester with AAV2, AAV2/1, HIV, or SIV-based vectors that indicate long-term transgene expression (up to 6 years) without evidence of adverse effects. These animals have exhibited stable expression in the muscular component of the diaphragm and peritoneum (1×10⁷–4×10⁹ photons/sec [p/s]) when using the prenatal systemic (intraperitoneal) ultrasound-guided approach for fetal gene transfer.