Inherited diseases of photoreceptors and prospects for gene therapy.
نویسندگان
چکیده
The photoreceptor cells of the retina are subject to a wide range of genetic diseases. This review summarizes current knowledge regarding an important group of retinal diseases caused by mutations in photoreceptor-enriched genes. In addition, progress toward treatment of a variety of these diseases in animal models via adeno-associated virus gene therapy is described. Although no human trials have yet been initiated to treat diseases caused by mutations in photoreceptor-enriched genes, there is a great deal of optimism regarding the prospects of treating these diseases using adeno-associated virus gene therapy.
منابع مشابه
نگاهی به ژن درمانی، پیشرفتهای اخیر و چشم انداز آینده
Human gene therapy has attracted increasing attention as a highly encouraging therapeutic approach to treat wide variety of diseases, other than genetically inherited and monogenic disorders. This approach entails the introduction and expression of a variety of nucleic acids into human target cells for therapeutic purposes. In this article, we review the history, highlights, recently progresses...
متن کامل[Gene therapy for inherited retinal dystrophies].
The inherited retinal dystrophies comprise a large number of disorders characterized by a slow and progressive retinal degeneration. They are the result of mutations in genes that express in either the photoreceptor cells or the retinal pigment epithelium. The mode of inheritance can be autosomal dominant, autosomal recessive, X linked recessive, digenic or mitochondrial DNA inherited. At the m...
متن کاملGene therapy for inherited retinal degeneration.
Research into the molecular and genetic basis of disease is continually expanding and improving the prospects for rational treatments. Of these, gene therapy (here defined as the introduction of genetic material into human cells) may ultimately oVer the greatest scope. This article provides a perspective on the potential for gene therapy for treating inherited retinal degenerations, along with ...
متن کاملGenes and Diseases
The science of medical genetics is progressing rapidly. The practical importance of this progress should be considered by medical doctors. Both genetic and environmental factors are involved in the abnormail phenotypes considered diseases. The relative significance of the two factors varies from disease to disease. All diseases can be viewed as falling on a spectrum in this regard. Hereditary...
متن کاملGene therapy to treat inherited and complex retinal degenerative diseases
These are exciting times in the field of retinal gene therapy, as impressive successes have been achieved by recombinant adenoassociated viral (rAAV) gene delivery of Rpe65 or Rep1 to the retinal pigment epithelium of patients with Lebers congential amaurosis or choroideremia, respectively.1–5 Instead of being left with incurable blindness, patients are reporting restoration of vision. An advan...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
عنوان ژورنال:
- Pharmacogenomics
دوره 9 3 شماره
صفحات -
تاریخ انتشار 2008