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A new strategy to enhance gene therapy for sickle cell diseaseAnne Galy, Ph.D., from Inserm, and co-authors, applied a novel lentivirus-based gene therapy strategy in CD34 + hematopoietic progenitor and stem cells (HSPCs) obtained from cord blood.
Results from the single-arm open-label trial have been able to elicit an extension of CD34+ cells ex vivo in two out of the 12 patients enrolled on the trial. The trial data, published in the New ...
With gene therapy, CD34+ cells can produce fetal haemoglobin, which then takes over from the faulty version. Yen carries out in vivo pharmacology and toxicology studies on these therapies in mice ...
We used oligonucleotide-based DNA microarrays to study global gene expression in ten AML+8 patients with +8 as the sole chromosomal abnormality and ten AML-CN patients, as well as seven CD34+ cell ...
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