[HTML][HTML] The X-linked retinopathies: physiological insights, pathogenic mechanisms, phenotypic features and novel therapies
X-linked retinopathies represent a significant proportion of monogenic retinal disease. They
include progressive and stationary conditions, with and without syndromic features. Many …
include progressive and stationary conditions, with and without syndromic features. Many …
Evaluation of dose and safety of AAV7m8 and AAV8BP2 in the non-human primate retina
PS Ramachandran, V Lee, Z Wei, JY Song… - Human gene …, 2017 - liebertpub.com
Within the next decade, we will see many gene therapy clinical trials for eye diseases, which
may lead to treatments for thousands of visually impaired people around the world. To target …
may lead to treatments for thousands of visually impaired people around the world. To target …
Inherited retinal degenerations: current landscape and knowledge gaps
Inherited retinal degenerations (IRDs) represent a diverse group of progressive, visually
debilitating diseases that can lead to blindness in which mutations in genes that are critical …
debilitating diseases that can lead to blindness in which mutations in genes that are critical …
Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters
Recombinant adeno-associated viral vectors (AAVs) are an effective system for gene
transfer. AAV serotype 2 (AAV2) is commonly used to deliver transgenes to retinal ganglion …
transfer. AAV serotype 2 (AAV2) is commonly used to deliver transgenes to retinal ganglion …
Let there be light: gene and cell therapy for blindness
Retinal degenerative diseases are a leading cause of irreversible blindness. Retinal cell
death is the main cause of vision loss in genetic disorders such as retinitis pigmentosa …
death is the main cause of vision loss in genetic disorders such as retinitis pigmentosa …
Optogenetic gene therapy for the degenerate retina: recent advances
ME McClements, F Staurenghi, RE MacLaren… - Frontiers in …, 2020 - frontiersin.org
The degeneration of light-detecting rod and cone photoreceptors in the human retina leads
to severe visual impairment and ultimately legal blindness in millions of people worldwide …
to severe visual impairment and ultimately legal blindness in millions of people worldwide …
rAAV-compatible MiniPromoters for restricted expression in the brain and eye
CN De Leeuw, AJ Korecki, GE Berry, JW Hickmott… - Molecular brain, 2016 - Springer
Background Small promoters that recapitulate endogenous gene expression patterns are
important for basic, preclinical, and now clinical research. Recently, there has been a …
important for basic, preclinical, and now clinical research. Recently, there has been a …
Advances in gene therapy for diseases of the eye
Over the last few years, huge progress has been made with regard to the understanding of
molecular mechanisms underlying the pathogenesis of neurodegenerative diseases of the …
molecular mechanisms underlying the pathogenesis of neurodegenerative diseases of the …
Shedding light on myopia by studying complete congenital stationary night blindness
C Zeitz, JE Roger, I Audo, C Michiels… - Progress in Retinal and …, 2023 - Elsevier
Myopia is the most common eye disorder, caused by heterogeneous genetic and
environmental factors. Rare progressive and stationary inherited retinal disorders are often …
environmental factors. Rare progressive and stationary inherited retinal disorders are often …
Dosage thresholds and influence of transgene cassette in adeno-associated virus–related toxicity
Today, there are> 500 published studies and 40 clinical trials to treat retinal disorders using
gene therapy. The great majority of them rely on the use of adeno-associated virus vectors …
gene therapy. The great majority of them rely on the use of adeno-associated virus vectors …