Researchers from the Salk Institute for Biological Studies have demonstrated for the first time ever that stem cell and gene therapy combination technique could work in human cells.
Previously several studies have conducted with mice but human studies were never successful. Though they did able to cure a cell not any diseased human but morning shows the day, still lot to go but if this studies really works then, if scientists could able to cure genetic problems through this combined technique the it will open up a door of limit less options.
Leader of the team Salk Professor Juan-Carlos Izpisua Belmonte and colleges, have corrected a defective gene in cells taken from patients with Fanconi anemia, a disease that can lead to bone marrow failure, leukemia and other cancers.
Researchers at first collected hair or skin cells from patients with Fanconi anemia, then the successfully corrected the gene defect and reprogram the cells into induced pluripotent stem cells – cells created synthetically from skin or other cells. Finally this FA-iPS cells were indistinguishable from human embryonic stem cells and iPS cells generated from healthy donors.
They used gene therapy techniques developed in the lab of Salk Professor Inder Verma, and also collaborated with researchers at the Center of Regenerative Medicine in Barcelona, Spain, and CIEMAT in Madrid, Spain.
Keeping in mind that Fanconi anemia always help bone marrow failure as a result of the progressive decline in the numbers of functional hematopoietic stem cells , researchers tested and found that FA-iPS cells readily differentiated into hematopoietic progenitor cells primed to differentiate into healthy blood cells.
Researchers are looking forward in particularly to over come the main obstacle that is preventing the reprogrammed cells from inducing tumors.
The study has been published online on Sunday by the journal Nature, offers the first proof that the technology can work in human cells – though more work remains for it to be tried in patients.
The researchers received a $6.6 million grant from California’s stem cell institute in April to continue the work. One big remaining problem is to prevent the reprogrammed cells from inducing tumors.