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Abstract

Abstract: DESCRIPTION (provided by applicant): The goal of the Phase I research is to develop ex vivo gene therapy that will achieve long-term, localized delivery of human neurotrophin-3 (hNT-3) in the rat contusion model of spinal cord injury. Our overall goal is to develop cell-based delivery of neurotrophins and other proteins as therapy for acute spinal cord injury. The following are the Phase I specific aims: Aim 1: To test functional recovery in response to NT-3 in rat spinal cord injury model. Aim 2: To test the effect of methylprednisotone on NT-3 based recovery of injured rats. In Phase II research we will develop a stereotaxic injection method to introduce cells into the injured spinal cords of rats, and study the effect of delaying implantation (24 to 48 hours post-injury) on cell survival, protein production, and functional recovery. Besides NT-3, we also will transduce cells with virus that produces brain-derived neurotrophic factor (BDNF), and look at its effect alone and in combination with NT-3 on functional recovery in animal models. Based on the results in Phase I we will continue working with methylprednisolone. We will test the system in a primate model, and develop human cells towards beginning Phase I clinical trials. PROPOSED COMMERCIAL APPLICATION: NOT AVAILABLE

Thesaurus Terms:
Sertoli cell, gene delivery system, gene targeting, gene therapy, methylprednisolone, neurotrophic factor, spinal cord injury, technology /technique development
Adenoviridae, cell transplantation
biotechnology, fluorescence microscopy, laboratory rat

Institution:	MANDALMED, INC.
	LAKESIDE MEDICAL CENTER
	SAN FRANCISCO, CA 94132
Fiscal Year:	2002
Department:
Project Start:	01-APR-2002
Project End:	31-MAR-2003
ICD:	NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT
IRG:	ZRG1