A treatment tested on rats combining gene reprogramming with ASC.
"They successfully used what is called a viral vector (in this case a lentivirus) to insert a healthy version of the IDUA gene into early stage red blood cell cultures"
"Encouraged by the initial cell experiments, the research team next cultured hematopoietic stem cells"
"Dr. Pan said reprogramming a patient's own developing red blood cells by gene therapy would provide a viable option for patients who cannot find a donor and avoid potential complications caused by an immune response to donor cells."
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