Abstract

The purpose of this study is to use an experimental form of gene therapy treatment for hemophilia, using a human Factor VIII retrovirus vector [hFVIII(V)]. In gene therapy, a gene is put into cells to provide different instructions from those provided by the genes a person already has. Genes are like """"""""blueprints"""""""" which provide detailed instructions to a cell about how to function. People with severe hemophilia are born without a gene, which makes normal FVIII. The goal of gene therapy for hemophilia is to provide a FVIII-producing gene to the body cells so that a person with hemophilia will be able to make his own FVIII. There are two primary goals of this study: 1) to find out how safe and effective human factor VIII vector treatment is for individuals with severe hemophilia A and 2) to measure the amount of Factor VIII which is produced in the body after a given dose of human factor VIII vector in order to determine the best dose for treatment.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR000046-40
Application #
6414071
Study Section
National Center for Research Resources Initial Review Group (RIRG)
Project Start
1974-10-01
Project End
2002-11-30
Budget Start
Budget End
Support Year
40
Fiscal Year
2000
Total Cost
Indirect Cost

  Institution

Name
University of North Carolina Chapel Hill
Department
Type
DUNS #
078861598
City
Chapel Hill
State
NC
Country
United States
Zip Code
27599

  Publications

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Hanly, John G; Li, Qiuju; Su, Li et al. (2018) Cerebrovascular Events in Systemic Lupus Erythematosus: Results From an International Inception Cohort Study. Arthritis Care Res (Hoboken) 70:1478-1487
Barber, Megan R W; Hanly, John G; Su, Li et al. (2018) Economic Evaluation of Lupus Nephritis in the Systemic Lupus International Collaborating Clinics Inception Cohort Using a Multistate Model Approach. Arthritis Care Res (Hoboken) 70:1294-1302
Rini, Christine; Vu, Maihan B; Lerner, Hannah et al. (2018) A qualitative study of patient and provider perspectives on using web-based pain coping skills training to treat persistent cancer pain. Palliat Support Care 16:155-169
Little, Jayne; Parker, Ben; Lunt, Mark et al. (2018) Glucocorticoid use and factors associated with variability in this use in the Systemic Lupus International Collaborating Clinics Inception Cohort. Rheumatology (Oxford) 57:677-687
Abdullah, Lubna H; Coakley, Raymond; Webster, Megan J et al. (2018) Mucin Production and Hydration Responses to Mucopurulent Materials in Normal versus Cystic Fibrosis Airway Epithelia. Am J Respir Crit Care Med 197:481-491
Martin, Maureen P; Naranbhai, Vivek; Shea, Patrick R et al. (2018) Killer cell immunoglobulin-like receptor 3DL1 variation modifies HLA-B*57 protection against HIV-1. J Clin Invest 128:1903-1912
Haas, David W; Bradford, Yuki; Verma, Anurag et al. (2018) Brain neurotransmitter transporter/receptor genomics and efavirenz central nervous system adverse events. Pharmacogenet Genomics 28:179-187

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