CORRECTION OF SEVERE COMBINED IMMUNO-DEFICIENCIES DUE TO V(D)J RECOMBINATION DEFECTS BY GENE THERAPY

Pubblicazioni Scientifiche

• BONE MARROW TRANSPLANTATION

  Hematopoietic stem cell transplantation in Omenn syndrome: a single-center experience

• CLINICAL IMMUNOLOGY

  Combined decrease of defined B and T cell subsets in a group of common variable immunodeficiency patients

• IMMUNOGENETICS

  Mutations of the T-cell receptor constant region after in utero stem cell transplantation

• JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

  Bone marrow transplantation for severe combined immune deficiency

• JOURNAL OF CLINICAL INVESTIGATION

  AIRE deficiency in thymus of 2 patients with Omenn syndrome

• JOURNAL OF CLINICAL INVESTIGATION

  A novel immunodeficiency associated with hypomorphic RAG1 mutations and CMV infection

• JOURNAL OF CLINICAL INVESTIGATION

  A hypomorphic R229Q Rag2 mouse mutant recapitulates human Omenn syndrome

• JOURNAL OF CLINICAL INVESTIGATION

  Gene therapy for severe combined immunodeficiency: are we there yet?

• JOURNAL OF PEDIATRICS

  Omenn syndrome in an infant with IL7RA gene mutation

• PEDIATRIC CARDIOLOGY

  Cardiac thrombus in Omenn syndrome