GENE TRANSFER DEVICE FOR AUTOLOGOUS BLOOD TRANSFUSION
DESCRIPTION (Adapted from applicant's abstract): This project will test the feasibility of using a new gene transfer device to transfect primary human blood progenitors, in vitro. The genetically modified blood precursor cells will be reintroduced by autologous transfusion and permitted to undergo terminal maturation in vivo. The transfected precursor cells will therefore serve as an effective blood substitute for patients with blood abnormalities. They will test this new device and approach using human sickle cell disease as a model. Each of the current sickle cell treatment procedures (heterologous transfusion, chemical inducers of fetal hemoglobin, and retroviral gene transfer) have significant deficiencies. For example, the possibility of infection with blood borne pathogens such as HIV haunts heterologous transfusion. The chemical agents used to induce the fetal globin gene have various side effects, and retroviral gene transfer, although efficient, suffers in the case of globin gene transfer from low expression and the ever present possibility of the formation of recombinant viruses. In Phase I, they are combining three new technologies to provide a novel approach to blood disorders. These are: (a) the ability to culture purified primary human erythroblasts, (b) particle-mediated DNA transfer (gene gun), and (c) a proprietary assay for transfection of erythroid cells which permits one to quantify and optimize the absolute efficiency of gene transfection. This approach will permit us, in Phase II or III, to introduce a fetal globin gene, in a construction which promotes high expression, into primary erythroid cultures of a sickle cell patient. These cells, expanded in culture, could be reintroduced into the patient ---i.e., autologous transfusion. $ = TOTAL AWARD AMTS & NOT LIMITED TO PORTION OF PROJECT RELATED TO SUBJECT OF SEARCH SUBPROJECT $ = TOTAL AWARD AMOUNT DIVIDED BY NUMBER OF SUBPROJECTS SOURCE: CRISP FORMAT F FY 97 LAST UPDATE 04-07-98 1QUERY 1536 ID SEARCH 06/01/98 PAGE 265 --PROJECT NUMBER......1 R43 DK52257-01 INVESTIGATOR NAME/ADDRESS FY 97 HOUCK, RAYMAND K IRG/INTRAMURAL UNIT..ZRG2 AUTOMATED CELL TECHNOLOGY, INC AWARD AMOUNT......... $100,000 832 12TH STREET OAKMONT, PA 15139 PERFORMING ORGANIZATION: AUTOMATED CELL TECHNOLOGY, INC. TITLE AUTOMATED BIOREACTOR FOR EXPANDING STEM CELLS ABSTRACT: The overall aim of this research program is to develop a single-cell bioreactor system that will automate the technology for culturing single cells and allow for the rapid selection and optimization of media and growth factors for stem cell growth. This system, which will allow cells to be grown in a sterile environment in which temperature, pH, and atmosphere (e.g., humidity and pO2) are regulated, will monitor continuously in situ the behavior of individual cells (e.g., division, differentiation, and motility) in multiple wells in a 96-well plate using a computer-controlled microscope imaging system with pattern recognition software to drive a robotic pipetting assembly to automatically change media in individual wells to respond to changes in cell number (growth) and phenotype (differentiation). We believe that this new technology will: 1) allow the reproducible growth of pluripotent stem cells; 2) allow automation of the study of cell growth, including the development of optimal substrates and growth factors for cell proliferation and differentiation, in a way that is superior in speed and efficiency compared to existing manual techniques; and 3) have widespread applications to many problems in cell biology, toxicology and tissue engineering.
Small Business Information at Submission:
Principal Investigator:Shapiro, steven g
4853 CORDELL AVE, STE 11A Bethesda, MD 20814
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