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Engineering Myoblasts for Transplantation

Award Information

Agency:
Department of Health and Human Services
Branch:
N/A
Award ID:
71022
Program Year/Program:
2004 / STTR
Agency Tracking Number:
HL078085
Solicitation Year:
N/A
Solicitation Topic Code:
N/A
Solicitation Number:
N/A
Small Business Information
BIOMEDICAL RESEARCH MODELS, INC.
67 MILLBROOK, ST, STE 422 WORCESTER, MA -
View profile »
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
 
Phase 1
Fiscal Year: 2004
Title: Engineering Myoblasts for Transplantation
Agency: HHS
Contract: 1R41HL078085-01
Award Amount: $223,141.00
 

Abstract:

DESCRIPTION (provided by applicant): The primary goals of stem cell biology are to develop practical cell-based tools: 1) to enhance the repair of damaged organs and tissues; and 2) as vehicles for gene therapy to introduce engineered genes to the body to either correct a genetic defect or to provide therapeutic molecules. The use of embryonic stem cells is both controversial and fraught with technical limitations that restrict their current use in a therapeutic setting. In contrast, the use of adult-derived, lineage-specific stem cells is currently the focus of several clinical trials. For example, two clinical trials are under way to assess the value of autologous muscle satellite cell transplantation to improve myocardial function following infarction. Since congestive heart failure accounts for more than 43% of Medicare's annual expenditures, the market for this therapy is substantial. Satellite cells are a resident population of muscle stem cells that can be harvested from virtually any patient with a simple needle biopsy. These cells can be expanded in vitro and then reintroduced in vivo to either repair tissues such as damaged myocardium or skeletal muscle, or instead engineered to express ectopic genes that are of therapeutic value. One of the major hurdles limiting the utility of myoblasts for transplantation is that relatively few of these cells survive past the first few days. We have identified a novel gene that plays a key regulatory role in the myoblast survival and differentiation in mammals. We have also demonstrated that expression of a dominant-negative form of this gene allows myoblasts to survive in the absence of trophic support, thus making it an ideal target for developing cell-based therapies. In this Phase I SBIR project, we will: 1) optimize methods for targeting the function of this protein in primary mouse myoblasts, and 2) determine if these engineered cells survive and contribute to muscle function in vivo.

Principal Investigator:

Edward H. Kislauskis
5084597544
EKISLAUSKIS@BIOMERE.COM

Business Contact:

Dennis Guberski
5084597544
DGUBERSKI@BIOMERE.COM
Small Business Information at Submission:

BIOMEDICAL RESEARCH MODELS, INC.
BIOMEDICAL RESEARCH MODELS, INC. 67 MILLBROOK, ST, STE 422 WORCESTER, MA 01606

EIN/Tax ID: 043338250
DUNS: N/A
Number of Employees: N/A
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
Research Institution Information:
UNIVERSITY OF MASSACHUSETTS AMHERST
GRANT & CONTRACT ADMINISTRATION
Amherst, MA 01003
RI Type: Nonprofit college or university