Results 151 to 160 of about 20,822 (186)
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2020
Gene therapy with autologous HSCs modified to express ß-globin represents a potential therapeutic option for transfusion-dependent beta-thalassemia. In order to investigate the safety and efficacy of lentiviral vector based gene therapy, we analyzed the viral integration profile in transduced HSCs using LAM-PCR and NGS method.
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Gene therapy with autologous HSCs modified to express ß-globin represents a potential therapeutic option for transfusion-dependent beta-thalassemia. In order to investigate the safety and efficacy of lentiviral vector based gene therapy, we analyzed the viral integration profile in transduced HSCs using LAM-PCR and NGS method.
openaire +1 more source
Hypertriglyceridemia in homozygous beta thalassemia
The Journal of Pediatrics, 1975A case of hypertriglyceridemia in association with homozygous beta-thalassemia in an 11-month-old female infant is reported. The hypertriglyceridemia proved to be secondary, as it was indicated by clinical features as well as laboratory findings. Attention is called to a possible alteration of lipid metabolism in association with thalassemia major.
M R, Ameri +3 more
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Birth defects original article series, 1988
In summary, the beta-thalassemias are due to defects in or around the structural beta-globin gene. In some Indian patients, there is deletion of sequence at the 3' end of the beta-globin gene. Most commonly, single nucleotide mutations cause beta(+)- and beta(0) -thalassemia. More than 30 such mutations have been identified.
A, Bank, C, Dobkin
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In summary, the beta-thalassemias are due to defects in or around the structural beta-globin gene. In some Indian patients, there is deletion of sequence at the 3' end of the beta-globin gene. Most commonly, single nucleotide mutations cause beta(+)- and beta(0) -thalassemia. More than 30 such mutations have been identified.
A, Bank, C, Dobkin
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The New England journal of medicine, 2005
Deborah, Rund, Eliezer, Rachmilewitz
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Deborah, Rund, Eliezer, Rachmilewitz
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Guidelines for Diagnosis and Management of Beta-Thalassemia Intermedia
Pediatric Hematology and Oncology, 2014Mehran Karimi +2 more
exaly
Marrow Transplantation in Patients with Thalassemia Responsive to Iron Chelation Therapy
New England Journal of Medicine, 1993Emanuele Angelucci +2 more
exaly