Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is caused by thymidine phosphorylase (TP) deficiency, which leads to toxic accumulations of thymidine (dThd) and deoxyuridine (dUrd). In this work, we report that infusion of platelets from healthy donors to patients with MNGIE restored transiently circulating TP and reduced plasma dThd and dUrd levels, suggesting that treatments to achieve permanent restoration of circulating TP such as allogeneic stem cell transplantation or gene transfer might be therapeutic.
The AutoPBSC is a reliable system for peripheral blood HPC collection in children mainly when used in combination with LVL. The major advantage of this software is a reduced need for priming. LVL allows better CD34+ cell collection efficiency, enhanced recruitment, and improved CD34+ cell yield.
Antibody elution by chloroform and trichloroethylene was compared with the Rubin ether method modified by Hughes-Jones for IgG antibodies and the Landsteiner and Miller heat method for IgM antibodies. After washing the cells, the chloroform and trichloroethylene method was done at 37 degrees C for 10 minutes. With this technique the eluate remained on the upper layer above the red blood cell stroma layer, while with Rubin's method the eluate remained below the cell stroma. The reported technique was as effective as the Landsteiner and Miller heat method for IgM antibodies, and a double volume of eluate was obtained.
Progenitor cell viability and function are preserved with this dry-thawing system. The time to hematopoietic engraftment of patients after transplantation is comparable to those infused with progenitor cells thawed with the water bath technique. Thawing cell products without the use of water and in a dry environment might favor the use of this dry method.
A survey of the results of laboratory investigations in 6 cases of drug‐induced hemolytic anemia showed that in 5 of them adsorption of immune complexes to red cells only occurred when the I antigen was expressed in the erythrocyte membrane.
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