<p>Adenosine deaminase (ADA) deficiency causes severe combined immunodeficiency (SCID) through an accumulation of toxic metabolites within lymphocytes. Recently, ADA deficiency has been successfully treated using lentiviral-transduced autologous CD34+ cells carrying  the ADA gene. T and B cell function appears to be fully restored but in many patients' B cell numbers remain low and assessments of the</p>

Gene Therapy Normalizes the IgH Repertoire in ADA Deficiency

Severe combined immunodeficiency due to adenosine deaminase deficiency study with RNA-Seq data on dbGaP (40 participants)

Gene Therapy Normalizes the IgH Repertoire in ADA Deficiency

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