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NOVEL EX VIVO GENE THERAPY FOR SPINAL CORD INJURY
Phone: (415) 333-5570
Email: ALPA@MANDALMED.COM
Phone: (415) 333-5570
Email: CONNIE@MANDALMED.COM
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
* Information listed above is at the time of submission. *