New research from the Advanced Gene and Cell Therapy Lab at Royal Holloway, University of London has used pioneering stem cell techniques to better understand why certain cells are more at risk of degenerating in Spinal Muscular Atrophy than others.
Dr Rafael Yáñez and his team from the School of Biological Sciences used Induced Pluripotent Stem Cells (iPSCs) which are created by treating cells from a skin biopsy, a hair follicle – or almost any body part – with chemicals and genes in the lab. The iPSCs were then ‘reprogrammed’ and coerced into forming motor neurons, the cells from the spinal cord which control movement and breathing.
The findings can now be used for testing therapies in the Induced Pluripotent Stem Cells and motor neurons.
Dr Rafael Yáñez, said: “We have observed a progressive reduction of SMN and PLS3 proteins during differentiation of induced pluripotent stem cells to motor neurons, similar to some reports in animals and some controversial data from human development. These changes may underpin the susceptibility of motor neurons to spinal muscular atrophy. We now will investigate the reason for the changes, and more importantly, progress our research for novel therapies taking these protein changes into account. The research also shows how we are trying to reduce reliance on animal experiments whenever possible, as these stem cells now allow the production of human cells that cannot be obtained directly from patients and we can study the cells in lab dishes.”
For this research Dr Yáñez collaborated with clinicians and scientists from the Hospital de la Santa Creu i Sant Pau, Barcelona, Spain, the Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Evry, France and the Beckman Research Institute, California, USA. The research in Dr Yáñez’s lab was funded by the Spanish government and the Spanish Spinal Muscular Atrophy Foundation FundAME (project GENAME), the 6th EU Framework Programme (project CLINIGENE) and Royal Holloway.”
Dr Rafael Yáñez and his team from the School of Biological Sciences used Induced Pluripotent Stem Cells (iPSCs) which are created by treating cells from a skin biopsy, a hair follicle – or almost any body part – with chemicals and genes in the lab. The iPSCs were then ‘reprogrammed’ and coerced into forming motor neurons, the cells from the spinal cord which control movement and breathing.
The findings can now be used for testing therapies in the Induced Pluripotent Stem Cells and motor neurons.
Dr Rafael Yáñez, said: “We have observed a progressive reduction of SMN and PLS3 proteins during differentiation of induced pluripotent stem cells to motor neurons, similar to some reports in animals and some controversial data from human development. These changes may underpin the susceptibility of motor neurons to spinal muscular atrophy. We now will investigate the reason for the changes, and more importantly, progress our research for novel therapies taking these protein changes into account. The research also shows how we are trying to reduce reliance on animal experiments whenever possible, as these stem cells now allow the production of human cells that cannot be obtained directly from patients and we can study the cells in lab dishes.”
For this research Dr Yáñez collaborated with clinicians and scientists from the Hospital de la Santa Creu i Sant Pau, Barcelona, Spain, the Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Evry, France and the Beckman Research Institute, California, USA. The research in Dr Yáñez’s lab was funded by the Spanish government and the Spanish Spinal Muscular Atrophy Foundation FundAME (project GENAME), the 6th EU Framework Programme (project CLINIGENE) and Royal Holloway.”
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