Reference of 4-Aminobutan-1-olIn 2021 ,《High-throughput evaluation of polymeric nanoparticles for tissue-targeted gene expression using barcoded plasmid DNA》 appeared in Journal of Controlled Release. The author of the article were Kim, Jayoung; Vaughan, Hannah J.; Zamboni, Camila G.; Sunshine, Joel C.; Green, Jordan J.. The article conveys some information:
Successful systemic gene delivery requires specific tissue targeting as well as efficient intracellular transfection. Increasingly, research laboratories are fabricating libraries of novel nanoparticles, engineering both new biomaterial structures and composition ratios of multicomponent systems. Yet, methods for screening gene delivery vehicles directly in vivo are often low-throughout, limiting the number of candidate nanoparticles that can be investigated. Here, we report a comprehensive, high-throughput method to evaluate a library of polymeric nanoparticles in vivo for tissue-specific gene delivery. The method involves pairing each nanoparticle formulation with a plasmid DNA (pDNA) that harbors a unique nucleotide sequence serving as the identifying “”barcode””. Using real time quant. PCR (qPCR) for detection of the barcoded pDNA and quant. reverse transcription PCR (RT-qPCR) for transcribed barcoded mRNA, we can quantify accumulation and transfection in tissues of interest. The barcode pDNA and primers were designed with sufficient sensitivity and specificity to evaluate multiple nanoparticle formulations per mouse, improving screening efficiency. Using this platform, we evaluated the biodistribution and transfection of 8 i.v. administered poly(beta-amino ester; PBAE) nanoparticle formulations, each with a PBAE polymer of differential structure. Significant levels of nanoparticle accumulation and gene transfection were observed mainly in organs involved in clearance, including spleen, liver, and kidneys. Interestingly, higher levels of transfection of select organs did not necessarily correlate with higher levels of tissue accumulation, highlighting the importance of directly measuring in vivo transfection efficiency as the key barcoded parameter in gene delivery vector optimization. To validate this method, nanoparticle formulations were used individually for luciferase pDNA delivery in vivo. The distribution of luciferase expression in tissues matched the transfection anal. by the barcode qPCR method, confirming that this platform can be used to accurately evaluate systemic gene delivery. In the experimental materials used by the author, we found 4-Aminobutan-1-ol(cas: 13325-10-5Reference of 4-Aminobutan-1-ol)
4-Aminobutan-1-ol(cas: 13325-10-5) is used in the synthesis of NSAIDs with anti-inflammatory properties. Also used in the synthesis of polyamine transport ligands with specificity against human cancers allowing easy access to specific cancer cells.Reference of 4-Aminobutan-1-ol
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