Abstract：
:The clinical success of gene replacement therapies in recent years has served as a proof of concept for the treatment of inherited retinal degenerations using adeno-associated virus (AAV) as viral vector. However, inherited retinal degenerative diseases showcase a broad genetic and mechanistic heterogeneity, challenging the development of mutation-specific therapies for each specific mutation. Mutation-independent approaches must be developed to slow down retinal degeneration regardless of the underlying genetic mutation and onset of the disease. New understanding of cell death mechanisms in rod-cone dystrophies have led to promising rescue of photoreceptor cell death by virally mediating expression of anti-apoptotic factors and secretion of retinal neurotrophic factors. Optogenetic therapies are also able to restore light sensitivities in blind retinas.
journal_name
Adv Exp Med Bioljournal_title
Advances in experimental medicine and biologyauthors
Fortuny C,Flannery JGdoi
10.1007/978-3-319-75402-4_10subject
Has Abstractpub_date
2018-01-01 00:00:00pages
75-81eissn
0065-2598issn
2214-8019journal_volume
1074pub_type
杂志文章,评审abstract：:Metabolism of fuels is driven by the energy demand of the organism and its regulation is influenced by many hormonal and metabolic factors. Insulin is of utmost importance in regulating glucose metabolism by promoting glucose uptake in the insulin-sensitive tissues for energy consumption and/or storage. The effects of...
journal_title：Advances in experimental medicine and biology
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journal_title：Advances in experimental medicine and biology
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doi：10.1007/0-387-24980-X_26
更新日期：2005-01-01 00:00:00
