Development of novel tissue directed ultra sound therapeutic gene delivery system

Award Information
Agency:
Department of Health and Human Services
Branch
n/a
Amount:
$718,896.00
Award Year:
2010
Program:
SBIR
Phase:
Phase II
Contract:
4R44HL095238-02
Award Id:
93947
Agency Tracking Number:
HL095238
Solicitation Year:
n/a
Solicitation Topic Code:
NHLBI
Solicitation Number:
n/a
Small Business Information
868 BAKER COURT, GLEN ELLYN, IL, -
Hubzone Owned:
N
Minority Owned:
N
Woman Owned:
N
Duns:
362147238
Principal Investigator:
ERIC COLES
() -
Business Contact:
STEVEN FEINSTEIN
() -
eric@sonogene.com
Research Institution:
n/a
Abstract
DESCRIPTION (provided by applicant): Low levels of high-density lipoprotein (HDL) cholesterol is an important risk factor for coronary heart disease (CHD). HDL cholesterol levels are predictive of major cardiovascular events in patients treated with statin s. This relationship was also observed among patients with LDL cholesterol levels below 70 mg per deciliter. Studies have demonstrated that polynucleotides, such as DNA and RNA, can be delivered to cells producing cellular changes that can be therapeutic. In order to successfully achieve gene therapy, it is essential that the genetic materials work in conjunction with an effective delivery vehicle to provide localized therapy. DNA coded to express a whole or partial protein can be delivered either directly to the organism, in situ, or indirectly by transfer to a cell that is then transplanted into the organism. SonoGene Corporation is researching the non-viral transfection of mammals as a means of gene therapy. It is proposed to develop a method for targetin g the transfection of DNA plasmid to the liver to enhance apoA-I expression. The request for this grant is to support research in developing a suitable non-invasive route to induce apoA-I gene expression in the liver for the purpose of favorably altering c holesterol metabolism. We propose to inject naked plasmid apoA-I DNA into the blood utilizing microspheres or microbubbles as a delivery vehicle. The capillary endothelial cells in the liver are fenestrated and permit enhanced uptake of plasmids into liver cells. The liver is then treated with ultrasound waves that disrupt the microspheres and cause transient porosity of liver endothelial cells, allowing for the uptake and expression of plasmid DNA by the liver. PUBLIC HEALTH RELEVANCE: High-density lipopro tein (HDL) cholesterol level is an important risk factor for coronary heart disease. Low HDL cholesterol levels are predictive of major cardiovascular events in patients. SonoGene proposes to develop novel ultrasound technology to raise HDL cholesterol lev els as a cardio-protective therapeutic strategy.

* information listed above is at the time of submission.

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