Titlebook: Biotechnologies for Gene Therapy; RNA, CRISPR, Nanobot Yang H. Yun,Kristine E. Yoder Book 2022 The Editor(s) (if applicable) and The Author

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https://doi.org/10.1007/978-3-642-72405-3e agent of adult T-cell leukemia/lymphoma (ATL), the immune-mediated neurodegenerative disease, HTLV-1-associated myelopathy/tropical spastic paraparesis (HAM/TSP), and a number of other inflammatory disorders. These HTLV-1-related diseases develop in a portion of infected individuals after a prolon

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https://doi.org/10.1007/978-3-7091-4738-2cells. In addition, retroviruses do not activate the immune system, allowing for multiple doses with the same viral vector. Retroviral vectors are also readily pseudotyped expanding their tropism to a variety of cell types. In spite of these advantages, gene therapy trials with gammaretrovirus murin

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https://doi.org/10.1007/978-3-7091-4738-2uctures combine several advantages, which make them well suited for numerous biomedical applications. Particular advantages of DNA nanostructures include unprecedented control over nanoscale geometry and precise functionalization with molecules including small molecule therapeutics, nucleic acids (e

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capsule

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Small Activating RNA Therapy for Angiogenesis,so become available for increasing gene expression. However, small RNAs have an advantage over longer RNAs for some purposes, such as cardiovascular gene therapy, as they are much more stable and induce fewer immunological responses. Vascular endothelial growth factor A, a key regulator of angiogene

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Muscular Dystrophy Therapy Using Viral Vector-based CRISPR/Cas,PR/Cas components. Efforts have been made to optimize the viral vector systems for efficient delivery of these components to treat DMD. Herein, we review diverse aspects of several viral vectors combined with CRISPR/Cas systems for DMD therapy and discuss their therapeutic potential and the challeng

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DNA Origami Nanodevices for Therapeutic Delivery Applications,n processes; briefly describe nonclinical applications; and focus on preclinical development of DNA nanostructures as drug delivery devices with an emphasis on nanostructures engineered via the DNA origami fabrication method.