Search Results - "Krämer, Chiara"
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Striated muscle-specific base editing enables correction of mutations causing dilated cardiomyopathy
Published in Nature communications (22-06-2023)“…Dilated cardiomyopathy is the second most common cause for heart failure with no cure except a high-risk heart transplantation. Approximately 30% of patients…”
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Lab-Scale Production of Recombinant Adeno-Associated Viruses (AAV) for Expression of Optogenetic Elements
Published in Methods in molecular biology (Clifton, N.J.) (2020)“…Optogenetics, that is, the use of photoswitchable/-activatable moieties to precisely control or monitor the activity of cells and genes at unprecedented…”
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Relevance of Assembly-Activating Protein for Adeno-associated Virus Vector Production and Capsid Protein Stability in Mammalian and Insect Cells
Published in Journal of virology (15-10-2017)“…The discovery that adeno-associated virus 2 (AAV2) encodes an eighth protein, called assembly-activating protein (AAP), transformed our understanding of…”
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Impact of the Assembly-Activating Protein on Molecular Evolution of Synthetic Adeno-Associated Virus Capsids
Published in Human gene therapy (01-01-2019)“…Over the last decade, the role of the assembly-activating protein (AAP) has begun to be dissected for the formation of adeno-associated virus (AAV) capsids…”
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Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders
Published in Science advances (23-09-2022)“…Bioengineering of viral vectors for therapeutic gene delivery is a pivotal strategy to reduce doses, facilitate manufacturing, and improve efficacy and patient…”
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