2007
DOI: 10.2174/156652307782793531
|View full text |Cite
|
Sign up to set email alerts
|

Neuroprotective Gene Therapy for the Treatment of Inherited Retinal Degeneration

Abstract: Inherited retinal degeneration (IRD) affects around 1/3000 of the population in Europe and the United States. It is a diverse group of conditions that results from mutations in any one of over 100 different genes. Many of the genes have now been identified and their functions elucidated, providing a major impetus to develop gene-based treatments. Whilst gene replacement and gene silencing strategies offer prospects for the treatment of specific inherited retinal disorders, other disorders may be less amenable … Show more

Help me understand this report

Search citation statements

Order By: Relevance

Paper Sections

Select...
2
1
1
1

Citation Types

0
13
0

Year Published

2008
2008
2018
2018

Publication Types

Select...
8
1

Relationship

0
9

Authors

Journals

citations
Cited by 26 publications
(13 citation statements)
references
References 62 publications
0
13
0
Order By: Relevance
“…Neuroprotective strategies do not target the specific cause of a disease but instead attempt to limit the consequences (i.e., degeneration of photoreceptors and deterioration of visual function) and may thus be applicable across a broad range of degenerative retinal disorders. During the last two decades, a number of neurotrophic factors (NFs) have been shown to attenuate photoreceptor degeneration and to partly preserve retinal function in a variety of animal models of hereditary retinal degeneration and other diseases involving photoreceptor loss [3, 10]. Because NFs normally have short half‐life times, do not ordinarily cross the blood‐retina barrier, and bear the risk of unacceptable side effects when administered systemically, strategies are being developed that permit a local and sustained delivery of these factors to the retina.…”
Section: Introductionmentioning
confidence: 99%
“…Neuroprotective strategies do not target the specific cause of a disease but instead attempt to limit the consequences (i.e., degeneration of photoreceptors and deterioration of visual function) and may thus be applicable across a broad range of degenerative retinal disorders. During the last two decades, a number of neurotrophic factors (NFs) have been shown to attenuate photoreceptor degeneration and to partly preserve retinal function in a variety of animal models of hereditary retinal degeneration and other diseases involving photoreceptor loss [3, 10]. Because NFs normally have short half‐life times, do not ordinarily cross the blood‐retina barrier, and bear the risk of unacceptable side effects when administered systemically, strategies are being developed that permit a local and sustained delivery of these factors to the retina.…”
Section: Introductionmentioning
confidence: 99%
“…A third possible approach is to apply retinal gene therapy as a means of expressing a protein that is not itself directly involved in the disease process, but which may have additional therapeutic benefits, such as neuroprotective proteins and growth factors 14 . This would potentially be broadly applicable to a wide range of inherited retinal degenerations independent of the specific genetic cause, which would have additional benefits for cases where there is no specific molecular diagnosis.…”
Section: Gain Loss and Augmentation Of Functionmentioning
confidence: 99%
“…A number of these factors have demonstrated roles in neuroprotection of various retinal cell types in vitro and when administered to mouse models of retinal degeneration [89, 98]. Brain-derived neurotrophic factor (BDNF) has been reported to prevent retinal ganglion and amacrine cell death [99–101] and can also rescue photoreceptor loss in animal models of retinal degeneration [102, 103].…”
Section: Epigenetic Mechanisms Of Neurotoxicitymentioning
confidence: 99%