Gene Therapy for Neurologic Disease

作者: S. T. Suhr , F. H. Gage

DOI: 10.1001/ARCHNEUR.1993.00540110122012

关键词: Ex vivoGenetic transferGenetic enhancementCancer researchExpression vectorNeurologic diseaseTransgeneBiologyImmunologyNervous systemIn vivo

摘要: Gene therapy is a potentially potent new method of treating number neurologic disorders previously considered refractory to current conventional therapeutic treatments. Numerous advances have been made in the construction expression vectors, cellular and viral transgene carriers, characterization target cells for neuronal gene therapy. Two primary approaches nervous system transfer emerged as result these advances. The vivo approach concentrates on direct genetic material using chemical agents. ex relies cultured that are subsequently implanted into host organism. Both methods used preliminary experiments designed test efficacy strategies amelioration dysfunction.

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