Abstract

A shared vector production facility is proposed which will supply clinical grade viral vector for the projects participating in this program. The core will also supply FDA and RAC required safety testing for patients participating in clinical gene therapy trials. A facility capable of producing and testing retroviral and adeno-associated viral vectors will be housed in the Cancer Research Building and will occupy 1800 sq. feet of space. The laboratory is specifically designed for the production of clinical grade viral vector under Good Laboratory and Good Manufacturing Practice criteria. Clinical grade supernate will be produced by a technician with experience in GMP production and a technician with experience in retroviral vector testing. The facility will be overseen by an investigator who has successfully written Investigational New Drug (IND) applications for retroviral vectors and is currently conducting clinical trials using retroviral vectors. The laboratory will play a critical role in bringing the viral vectors developed in this project to clinical trial.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Research Program Projects (P01)
Project #
5P01HL053586-05
Application #
6110410
Study Section
Project Start
1998-12-01
Project End
2000-03-14
Budget Start
1998-10-01
Budget End
1999-09-30
Support Year
5
Fiscal Year
1999
Total Cost
Indirect Cost

  Institution

Name
Indiana University-Purdue University at Indianapolis
Department
Type
DUNS #
005436803
City
Indianapolis
State
IN
Country
United States
Zip Code
46202

  Publications

Stein, Stefan; Scholz, Simone; Schwäble, Joachim et al. (2013) From bench to bedside: preclinical evaluation of a self-inactivating gammaretroviral vector for the gene therapy of X-linked chronic granulomatous disease. Hum Gene Ther Clin Dev 24:86-98
Song, Liujiang; Kauss, M Ariel; Kopin, Etana et al. (2013) Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo. Cytotherapy 15:986-98
Liu, Ying; Ballman, Kimberly; Li, Deqiang et al. (2012) Impaired function of Fanconi anemia type C-deficient macrophages. J Leukoc Biol 91:333-40
Ou, Xuan; Chae, Hee-Don; Wang, Rui-Hong et al. (2011) SIRT1 deficiency compromises mouse embryonic stem cell hematopoietic differentiation, and embryonic and adult hematopoiesis in the mouse. Blood 117:440-50
Grez, Manuel; Reichenbach, Janine; Schwäble, Joachim et al. (2011) Gene therapy of chronic granulomatous disease: the engraftment dilemma. Mol Ther 19:28-35
Rohrabaugh, Sara L; Campbell, Timothy B; Hangoc, Giao et al. (2011) Ex vivo rapamycin treatment of human cord blood CD34+ cells enhances their engraftment of NSG mice. Blood Cells Mol Dis 46:318-20
Broxmeyer, Hal E; Lee, Man-Ryul; Hangoc, Giao et al. (2011) Hematopoietic stem/progenitor cells, generation of induced pluripotent stem cells, and isolation of endothelial progenitors from 21- to 23.5-year cryopreserved cord blood. Blood 117:4773-7
Rohrabaugh, Sara L; Hangoc, Giao; Kelley, Mark R et al. (2011) Mad2 haploinsufficiency protects hematopoietic progenitor cells subjected to cell-cycle stress in vivo and to inhibition of redox function of Ape1/Ref-1 in vitro. Exp Hematol 39:415-23
Liu, Ying; Timani, Khalid; Mantel, Charlie et al. (2011) TIP110/p110nrb/SART3/p110 regulation of hematopoiesis through CMYC. Blood 117:5643-51
Hawkins, Troy B; Dantzer, Jessica; Peters, Brandon et al. (2011) Identifying viral integration sites using SeqMap 2.0. Bioinformatics 27:720-2

Showing the most recent 10 out of 158 publications