Abstract

There is no standard therapy for sickle cell disease or thalassemia. Some patients continued expression of small amounts of fatal globin has completely prevented clinical disease in these disorders, suggesting that, continued expression of the adult fetal globin gene after birth will prevent manifestations of disease. Chemotherapeutic agents are toxic in children.
The aim of this proposal is to apply a new class of compound, isobutyramide an analogue of butyric acid to reactivate the fetal globin gene expression.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR000533-28
Application #
5220616
Study Section
Project Start
Project End
Budget Start
Budget End
Support Year
28
Fiscal Year
1996
Total Cost
Indirect Cost

  Publications

Christensen, Kurt D; Uhlmann, Wendy R; Roberts, J Scott et al. (2018) A randomized controlled trial of disclosing genetic risk information for Alzheimer disease via telephone. Genet Med 20:132-141
Kattan, Meyer; Bacharier, Leonard B; O'Connor, George T et al. (2018) Spirometry and Impulse Oscillometry in Preschool Children: Acceptability and Relationship to Maternal Smoking in Pregnancy. J Allergy Clin Immunol Pract 6:1596-1603.e6
Rhee, Rennie L; Davis, John C; Ding, Linna et al. (2018) The Utility of Urinalysis in Determining the Risk of Renal Relapse in ANCA-Associated Vasculitis. Clin J Am Soc Nephrol 13:251-257
Liebschutz, Jane M; Buchanan-Howland, Kathryn; Chen, Clara A et al. (2018) Childhood Trauma Questionnaire (CTQ) correlations with prospective violence assessment in a longitudinal cohort. Psychol Assess 30:841-845
Guan, Yue; Roter, Debra L; Wolff, Jennifer L et al. (2018) The impact of genetic counselors' use of facilitative strategies on cognitive and emotional processing of genetic risk disclosure for Alzheimer's disease. Patient Educ Couns 101:817-823
Altman, Matthew C; Whalen, Elizabeth; Togias, Alkis et al. (2018) Allergen-induced activation of natural killer cells represents an early-life immune response in the development of allergic asthma. J Allergy Clin Immunol 142:1856-1866
Berti, Alvise; Warner, Roscoe; Johnson, Kent et al. (2018) Brief Report: Circulating Cytokine Profiles and Antineutrophil Cytoplasmic Antibody Specificity in Patients With Antineutrophil Cytoplasmic Antibody-Associated Vasculitis. Arthritis Rheumatol 70:1114-1121
Wallace, Zachary S; Miloslavsky, Eli M; Cascino, Matthew et al. (2017) Effect of Disease Activity, Glucocorticoid Exposure, and Rituximab on Body Composition During Induction Treatment of Antineutrophil Cytoplasmic Antibody-Associated Vasculitis. Arthritis Care Res (Hoboken) 69:1004-1010
Gern, James E; Calatroni, Agustin; Jaffee, Katy F et al. (2017) Patterns of immune development in urban preschoolers with recurrent wheeze and/or atopy. J Allergy Clin Immunol 140:836-844.e7
Guan, Yue; Roter, Debra L; Erby, Lori H et al. (2017) Disclosing genetic risk of Alzheimer's disease to cognitively impaired patients and visit companions: Findings from the REVEAL Study. Patient Educ Couns 100:927-935

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