2018•Case Medical ResearchRequires access

Orphan designation: Autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene, Treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency

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Abstract

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About this research paper

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Available abstract

Key concepts: Adenosine deaminase, Adenosine deaminase deficiency, Severe combined immunodeficiency, Genetic enhancement, Viral vector, Transfection, Vector (molecular biology), Immunodeficiency