DURHAM, N.C., Dec. 17, 2015 /PRNewswire-iReach/ -- A new study appearing in STEM CELLS Translational Medicine shows that ground-state, patient-specific induced pluripotent stem cells show significant promise for disease modeling, gene editing and future clinical therapy.
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Ultimately, the discovery by a Chinese research team could lead to improved treatment for genetic diseases such as beta thalassemia, an inherited blood disorder caused by flawed or missing genes.
Scientists conducting the study derived induced pluripotent stem cells (iPSCs) in a ground, or naïve, state of pluripotency from non-reproductive cells of patients with beta thalassemia and injected them into mice embryos. They then examined how well those stem cells performed compared to non-naïve stem cells derived from the same non-reproductive cells.
They learned that the naïve cells functioned just as well as the others in terms of proliferation, gene expression and the ability to change into progenitor cells and did an even better job of correcting damaged genes.
“The single-cell cloning efficiency of the naïve iPSCs reached up to 88 percent, far higher than the efficiency of the primed iPSCs,” they said. “These results indicate that naive iPSC lines can be successfully generated from beta thalassemia fibroblasts under defined culture conditions.”
So far, there has been no effective treatment for beta thalassemia, a disorder also known as Cooley’s anemia. People with the condition often must undergo frequent blood transfusions, a therapy that causes iron to build up in the body over time and can bring about heart failure as early as the teens or early 20s.
Besides generating naïve iPSCs from human non-reproductive cells, the Chinese scientists also generated the cells from human urine samples, a step that could not only lead to a better, less invasive treatment for beta thalassemia but also give researchers an improved way to obtain stem cells for future studies, they said.
“The emergence of iPSC technologies and the development of gene-targeting strategies bring new hope for treating genetic diseases,” they said. “Our findings demonstrate the feasibility and superiority of using patient-specific iPSCs in the naïve state, which has important implications for clinical use of human iPSCs in the future.”
A dozen researchers at Tongji University in Shanghai and Guangzhou Medical University collaborated on the study supported by China’s National Natural Science Foundation and Ministry of Science and Technology, Shanghai’s Science and Technology and Education commissions and other sources.
“This strategy for reprogramming skin cells and even cells from urine -- into their “ground state” resulted in cells with high gene-correction efficiencies, suggesting their promise for future clinical therapy,” Anthony Atala, M.D., Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.
The full article, “Naïve Induced Pluripotent Stem Cells Generated From -Thalassemia Fibroblasts Allow Efficient Gene Correction with CRISPR/Cas9,” can be accessed at http://stemcellstm.alphamedpress.org/content/early/2015/12/14/sctm.2015-0157.full.pdf+html.
About STEM CELLS Translational Medicine: STEM CELLS Translational Medicine (SCTM), published by AlphaMed Press, is a monthly peer-reviewed publication dedicated to significantly advancing the clinical utilization of stem cell molecular and cellular biology. By bridging stem cell research and clinical trials, SCTM will help move applications of these critical investigations closer to accepted best practices.
About AlphaMed Press: Established in 1983, AlphaMed Press with offices in Durham, NC, San Francisco, CA, and Belfast, Northern Ireland, publishes two other internationally renowned peer-reviewed journals: STEM CELLS® (www.StemCells.com is the world’s first journal devoted to this fast paced field of research. The Oncologist® (www.TheOncologist.com), also a monthly peer-reviewed publication, is devoted to community and hospital-based oncologists and physicians entrusted with cancer patient care. All three journals are premier periodicals with globally recognized editorial boards dedicated to advancing knowledge and education in their focused disciplines.
Media Contact:Sharon Lee, AlphaMed Press, 9196800011, sharonlee@alphamedpress.com
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SOURCE STEM CELLS Translational Medicine
