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Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.

Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity. Sci Transl Med. 2014 Mar 12; 6(227):227ra33.

View in: PubMed

subject areas
  • Adolescent
  • Animals
  • Blood Platelets
  • Child
  • Child, Preschool
  • Clone Cells
  • Colitis
  • Disease Progression
  • Genetic Therapy
  • Hematopoietic Stem Cell Transplantation
  • Hematopoietic Stem Cells
  • Humans
  • Leukocytes, Mononuclear
  • Lymphocytes
  • Mice
  • Mice, Inbred NOD
  • Mutagens
  • Precursor T-Cell Lymphoblastic Leukemia-Lymphoma
  • Thrombocytopenia
  • Transplantation, Autologous
  • Treatment Outcome
  • Wiskott-Aldrich Syndrome
  • Wiskott-Aldrich Syndrome Protein

authors with profiles
  • Miguel Raul Abboud