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SYMPOSIUM THEME
Hemoglobin S - Making an Evolutionary Perfected Process, Imperfect 

Journal of Sickle Cell Disease and Hemoglobinoopathies
SYMPOSIUM ABSTRACTS

Meeting Location:

Greater Ft. Lauderdale Convention Center
1950 Eisenhower Blvd, Fort Lauderdale, Florida, USA  33316
April 28 - 30, 2017 
Website:  fscdr.org
Email:  symposium@fscdr.org
Is this a CME Medical Meeting?  Yes
 
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avatar for Osheiza Abdulmalik, Ph.D.

Osheiza Abdulmalik, Ph.D.

The Children's Hospital of Philadelphia, Research Institute
Research Associate II, Rivella Laboratory

Dr. Abdulmalik’s research interests broadly encompass the hemoglobinopathies and red blood cell disorders, with particular focus on sickle cell disease and beta-thalassemia. Though both disorders are consequences of simple genetic mutations, the implications of these mutations are inherently complex, and include significant morbidities, lifelong challenges, reduced quality of life, and early mortality in patients; as well as substantial healthcare burden on families, caregivers, and society in general. Dr. Abdulmalik’s current research focuses on three modalities of countering disease pathophysiology. The first approach, in tandem with a multidisciplinary team of collaborators, utilizes medicinal chemistry and structural biology to design novel drugs that block the primary pathophysiology of sickle cell disease, i.e., the polymerization of sickle hemoglobin. He employs an array of in vitro and in vivo assays to investigate the efficacy and pharmacokinetic/pharmacodynamic properties of select candidate drugs. The second approach investigates targeted mutations in the 3’ untranslated region of the beta globin gene to enhance the stability of the mRNA, with the ultimate goal of improving transgenes for gene therapy. The third approach investigates mutations in the coding region of the therapeutic beta-globin gene variants, which would, in addition to replacing the defective gene, confer additional beneficial effects, such as countering hemoglobin polymerization.