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The β-haemoglobinopathies are the most prevalent inherited disorders worldwide. Gene therapy of β-thalassaemia is particularly challenging given the requirement for massive haemoglobin production in a lineage-specific manner and the lack of selective advantage for corrected haematopoietic stem cells. Compound β(E)/β(0)-thalassaemia is the most common form(More)
X-linked severe-combined immunodeficiency (SCID-X1) has been treated by therapeutic gene transfer using gammaretroviral vectors, but insertional activation of proto-oncogenes contributed to leukemia in some patients. Here we report a longitudinal study of gene-corrected progenitor cell populations from 8 patients using 454 pyrosequencing to map vector(More)
The effects of trehalose pretreatment on thylakoid membranes of winter wheat were investigated under heat stress. Under normal growth conditions, the winter wheat synthesized 502 μg g−1(f.m.) trehalose, which increased to 1250 μg g−1(f.m.) under heat stress and to 1658 μg g−1(f.m.) in trehalose-pretreated seedlings. Under heat stress, proteins in the(More)
Clostridium difficile infection (CDI) causes nearly half a million cases of diarrhea and colitis in the United States each year. Although the importance of the gut microbiota in C. difficile pathogenesis is well recognized, components of the human gut flora critical for colonization resistance are not known. Culture-independent high-density Roche 454(More)
We investigated the drought resistance of a wheat (Triticum aestivum L.) stay-green mutant tasg1 and its wild-type (WT) in field experiments conducted for two years. Drought stress was imposed by controlling irrigation and sheltering the plants from rain. Compared with the WT, tasg1 exhibited a distinct delayed senescence under both normal and drought(More)
Hepatitis C virus (HCV) replication in infected patients produces large and diverse viral populations, which give rise to drug-resistant and immune escape variants. Here, we analyzed HCV populations during transmission and diversification in longitudinal and cross-sectional samples using 454/Roche pyrosequencing, in total analyzing 174,185 sequence reads.(More)
To investigate the role of glycine betaine in photosynthesis under stress, a transgenic wheat (Triticum aestivum L.) line T6 overaccumulating glycine betaine and its wild type Shi4185 were used. Seedlings were exposed to conditions of drought (30%, PEG-6000), heat (40°C) and their combination. The results revealed ultrastructural damage to the chloroplast(More)
BACKGROUND The outcomes of gene therapy to correct congenital immunodeficiencies are unknown. We reviewed long-term outcomes after gene therapy in nine patients with X-linked severe combined immunodeficiency (SCID-X1), which is characterized by the absence of the cytokine receptor common gamma chain. METHODS The nine patients, who lacked an HLA-identical(More)
Using isolated Alzheimer neurofibrillary tangles as the immunogen, nine mouse hybridomas were generated which produced antibodies to the tangles as tested in both tissue sections and isolated neurons from Alzheimer brain. Extraction of isolated neurofibrillary tangles with 2% SDS could not remove the antigen(s) with which these monoclonal antibodies(More)
Previously, several individuals with X-linked SCID (SCID-X1) were treated by gene therapy to restore the missing IL-2 receptor gamma (IL2RG) gene to CD34+ BM precursor cells using gammaretroviral vectors. While 9 of 10 patients were successfully treated, 4 of the 9 developed T cell leukemia 31-68 months after gene therapy. In 2 of these cases, blast cells(More)