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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.
Oyun teknolojileri kapsamında Türkiye’de 2021 yılında faaliyet sunmaya başlayan SloganBahis, temel vizyonu ile çalışmalarını sürdüren bir eğlence işletmecisidir. Yüksek güvenlik standartları ve teknik açıdan sorunsuz dijital oyun ortamı sunmasıyla sektördeki yerini almıştır.
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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Metachromatic Leukodystrophy Treatment Market Outlook 2034 The global industry was valued at US$ 2.5 Bn in 2023 It is expected to grow at a CAGR of 5.6% from 2024 to 2034 and reach US$ 4.5 Bn by the end of 2034
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Our journey began in 2009 when we established ourselves as CBO Infotech, operating as a proprietorship. Over the years, our company has continually evolved and undergone significant transformations. In subsequent years, we progressed to become CBO Infotech Pvt Limited, In 2021 we rebranded ourselves as CBO ERP Limited, Today we proudly serving 1100+ customer base in Bharat and overseas.https://www.cboerp.com/latest-news.php
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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