Scientists from Australia have developed a new way to perform stem cell gene correction, a process where stem cells are reprogrammed and genetically corrected.
The researchers, from the Morgridge Institute for Research and the Murdoch Children’s Research Institute (MCRI), say the method is dramatically faster than previous methods.
The study shows that genetically repaired stem cells can be created from a patient’s skin cells in as little as two weeks, compared to older approaches which took more than three months.
This innovative new technique combined two essential steps in preparing stem cells to achieve the extra speed. Firstly, the adult cells must be reprogrammed to become Induced Pluripotent Stem Cells (IPSCs). IPSCs are similar to embryonic stem cells and can differentiate into 220 types of cells in the human body. Then, those IPSCs must undergo a gene-editing process to remove whatever genetic mutation they may have. Combining those two steps saves a huge amount of time.
The study was led by Sara Howden, a postdoctoral fellow at MCRI and formerly with the Morgridge Institute. It was tested successfully on a patient with retinal degeneration and on an infant patient with immunodeficiency.
Howdy describes the benefit of the new method, saying: “The method developed in our study could potentially advance transplant medicine by making gene-corrected cells available to patients in a much timelier manner, and at a lower cost. It will have implications immediately for researchers working in regenerative medicine.”
This new process will make it much easier to conduct patient-specific IPS cell studies, because the timeline has been greatly sped up. The approach uses the new Cas9/CRISPR technology to combine the two steps.
The new process is expected to also be of great use in the development of personalised medicines — where a person’s specific genetic makeup and disease is analysed to create a unique treatment.
This new stem cell gene correction is an important step which will make many future therapies possible.
Source: New stem cell gene correction process puts time on researchers’ side
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