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