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- W2511461874 abstract "Gene therapy and cell therapy are intersecting fields of biomedical research with the common aim of repairing the direct causes or consequences of genetic diseases by using genes and cells, respectively. Gene therapy is broadly defined as a set of strategies that modify the expression of an individual's genes for therapeutic benefit. It requires the administration of a specific deoxyribonucleic acid (DNA) (or ribonucleic acid (RNA)) into the patient's cells. Cell therapy on the other hand requires administration of a population of intact live cells to a patient whose cells have been lost due to injury or disease. These emerging strategies can also be applied in acquired diseases in order to re-establish equilibrium and halt disease progression. In some cases, gene and cell therapy may be combined in order to obtain a therapeutic effect.In addition to becoming emerging therapeutics in the clinic, these two fields of research continually generate tools, concepts and techniques for elucidating biological questions across multiple disciplines.Key Concepts The retina lines the back of the eye and comprises six different types of neuronal cells and their underlying retinal pigment epithelium (RPE).Photoreceptors are the light-sensitive first-order neurons of the retina which capture the light stimulus and transform it into an electric signal.Human iPSC have the same features of hESC in terms of their differentiation capacity.Gene therapy is the use of genetic material as a drug to treat a chronic condition.Keywords:cell therapy;gene therapy;human-induced pluripotent stem cell;human embryonic stem cell;stem-cell derived RPE;stem cell-derived retinal progenitor;photoreceptor;viral vectors;genetics;retina" @default.
- W2511461874 created "2016-09-16" @default.
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- W2511461874 date "2016-08-16" @default.
- W2511461874 modified "2023-10-16" @default.
- W2511461874 title "Gene and Cell Therapy for Inherited Retinal Dystrophies" @default.
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- W2511461874 doi "https://doi.org/10.1002/9780470015902.a0026565" @default.
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