Abstract

The purpose of this research protocol is to test whether we can safely introduce the normal Fanconi Anemia group A (FAA) gene into hematopoietic stem cells of patients with this disease. This is to determine if a virus containing a normal FAA gene can correct the growth of the blood cells. Fanconi's anemia (FA) is an inherited disorder that can produce bone marrow failure. Some patients with FA have physical defects, usually involving the skeleton or kidneys. The major problem for most patients is aplastic anemia. In aplastic anemia, the blood counts for the red cells, white blood cells, and platelets are low because the bone marrow fails to produce these cells. Some patients with FA also can develop leukemia or cancers of other organs. The major purpose of this protocol is to test the safety of this technique and to determine whether we can transfer the FAA gene successfully.

  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 #
6414037
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

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
Venuto, Charles S; Lim, Jihoon; Messing, Susan et al. (2018) Inflammation investigated as a source of pharmacokinetic variability of atazanavir in AIDS Clinical Trials Group protocol A5224s. Antivir Ther 23:345-351
Malinen, Melina M; Kauttonen, Antti; Beaudoin, James J et al. (2018) Novel In Vitro Method Reveals Drugs that Inhibit Solute Transporter Alpha/Beta (OST?/?). Mol Pharm :
Li, Binglan; Verma, Shefali S; Veturi, Yogasudha C et al. (2018) Evaluation of PrediXcan for prioritizing GWAS associations and predicting gene expression. Pac Symp Biocomput 23:448-459
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

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