Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome
Cells control new filament assembly through the induction of nucleation promoting factors such as the WASp/WAVE (Wiskott-Aldrich syndrome
protein/WASp-family verprolin-homologous protein) family proteins.
(1,3,4) Interestingly, it has been revealed that LCA also has an association with different congenital and immunological conditions, such as inflammatory bowel disease, (1) Wiskott-Aldrich syndrome
, (13) Epstein syndrome, (14) lymphocytic colitis, SLE, (15) ankylosing spondylitis, psoriasis, (16) Gaucher's disease, (17) myelodysplastic syndrome, (9) chronic glomerulonephritis, and aplastic anemia.
The sixteen lines of stem cells on the NIH Registry carry genes for various hereditary disorders including Duchenne muscular dystrophy, Huntington's disease, cystic fibrosis, and rarer conditions such as Von Hippel-Lindau Syndrome, Wiskott-Aldrich syndrome
, spinal muscular atrophy, myotonic dystrophy and neurofibromatosis.
In one study, three boys with Wiskott-Aldrich syndrome
, an inherited disease that disables the immune system, received gene therapy.
The Aids virus has been used to cure two severe hereditary diseases - metachromatic leukodystrophy and Wiskott-Aldrich syndrome
Giant atypical MC lesions can be seen in patients with atopic dermatitis, those on corticosteroid or immunosuppressive therapy, sarcoidosis, leukaemia, Wiskott-Aldrich syndrome
, and AIDS.3-5 The giant lesion is rarely seen in healthy individuals.
One-step surgical approach of a thoracic aortic aneurysm in Wiskott-Aldrich syndrome
. Ann Thorac Surg 2007; 83:1537-8.
Therefore, Evans syndrome (ES) should be presented separetly then autoimmune hemolytic anemia (AIHA) as of Wiskott-Aldrich syndrome
(WAS) in which Coombs positivity may develop as of alloimmune reaction.
* Thrombocytopenia and the presence of microthrombocytes in male infants suggest Wiskott-Aldrich syndrome
Prior to the AIDS epidemic, PML was rarely seen in other immunocompromised patients occurring usually as a terminal event such as in cases of leukemia, lymphoma, systemic lupus erythematosus (SLE), Wiskott-Aldrich syndrome
, and severe combined immunodeficiency (SCID).
The remaining 2 samples were from infants diagnosed with Omenn syndrome and Wiskott-Aldrich syndrome
. The affected infants had lower estimated T-cell counts than controls.