Titlebook: Retinal Degenerative Diseases; Mechanisms and Exper Catherine Bowes Rickman,Christian Grimm,Joe G. Hol Conference proceedings 2019 Springer

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Small Molecule-Based Inducible Gene Therapies for Retinal Degenerationpotential gene therapies introduced into the eye can be evaluated by noninvasive methods such as fundoscopy, electroretinography, and optical coherence tomography. In the last two decades, great advances have been made in understanding the molecular underpinnings of retinal degenerative diseases. Bu

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同位素

A Comparison of Inducible Gene Expression Platforms: Implications for Recombinant Adeno-Associated V for autosomal dominant or complex retinal diseases, where uncontrolled transgene overexpression may lead to deleterious off-target effects and accelerated disease progression. While numerous platforms exist that allow for modulation of gene expression levels – ranging from inducible promoters to de

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Emerging Concepts for RNA Therapeutics for Inherited Retinal Diseasee expression on an RNA level have significant promise for treating IRD. In this review, we review the molecular basis of oligonucleotide therapeutics such as ribozymes, RNA interference (RNAi), antisense oligonucleotides (ASO), CRISPRi/a, and their applications to treatments of IRD.

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Conference proceedings 2019d posters at the meeting contributed to this volume. The blinding diseases of inherited retinal degenerations have no treatments, and age-related macular degeneration has no cures, despite the fact that it is an epidemic among the elderly, with 1 in 3-4 affected by the age of 70. The RD Symposium fo

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https://doi.org/10.1007/978-3-030-27378-1animal models; degneration; oxidative stress; carotenoids; gene therapy; protective therapy; photoreceptor