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Following the implementation of next-generation sequencing technologies (e.g., exome and genome sequencing) in molecular diagnostics, the majority of genetic defects underlying inherited retinal disease (IRD) can readily be identified. In parallel, opportunities to counteract the molecular consequences of these defects are rapidly emerging, providing hope for personalized medicine. ‘Classical’ gene augmentation therapy has been under study for several genetic subtypes of IRD and can be considered a safe and sometimes effective therapeutic strategy. The recent market approval of the first retinal gene augmentation therapy product (LuxturnaTM, for individuals with bi-allelic RPE65 mutations) by the FDA has not only demonstrated the potential of this specific approach, but also opened avenues for the development of other strategies. However, every gene—or even every mutation—may need a tailor-made therapeutic approach, in order to obtain the most efficacious strategy with minimal risks associated. In addition to gene augmentation therapy, other subtypes of molecular therapy are currently being designed and/or implemented, including splice modulation, DNA or RNA editing, optogenetics and pharmacological modulation. In addition, the development of proper delivery vectors has gained strong attention, and should not be overlooked when designing and testing a novel therapeutic approach. In this Special Issue, we aim to describe the current state of the art of molecular therapeutics for IRD, and discuss existing and novel therapeutic strategies, from idea to implementation, and from bench to bedside.

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alex vein

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Microalgae are photosynthetic organisms with the ability to sequester and convert atmospheric carbon dioxide into high-value bioactives, and are therefore seen as a renewable and sustainable bioresource in the fields of biofuels, aquaculture and animal feeds, bioremediation of waste, nutraceuticals, pharmaceuticals, cosmeceuticals and agriculture. Moreover, algae can adjust their metabolism according to surrounding growth conditions, and this metabolic flexibility can be exploited in industrial biotechnology with genetic and metabolic engineering, when compared to other photosynthetic organisms. The metabolome is the result of the combined effects of genetic and environmental influences on metabolic processes. Metabolomic studies can provide a global view of metabolism and thereby improve our understanding of the underlying biology. Advances in metabolomic technologies have shown utility for elucidating the mechanisms which underlie fundamental biological processes, including disease pathology. This book represents research papers based around metabolomics, to improve knowledge on the metabolome and metabolism in algae, with a focus on carbon and nitrogen resource allocation. It also describes many bioanalytical techniques and emphasizes their usefulness in microalgal biotechnology. Other aspects from an ecological, biotechnological and waste-water remediation perspective are also covered.

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Discover the economic impact of GLP-1 on U.S. consumer markets, from portion shifts and retail resets to demand forecasting strategy.

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Edgar Thurston

PD-US

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