Marchesi, Elena; Bovolenta, Matteo; Preti, Lorenzo; Capobianco, Massimo L.; Mamchaoui, Kamel; Bertoldo, Monica; Perrone, Daniela published an article in 2021. The article was titled 《Synthesis and Exon-Skipping Properties of a 3′-Ursodeoxycholic Acid-Conjugated Oligonucleotide Targeting DMD Pre-mRNA: Pre-Synthetic versus Post-Synthetic Approach》, and you may find the article in Molecules.Computed Properties of C6H15NO The information in the text is summarized as follows:
Steric blocking antisense oligonucleotides (ASO) are promising tools for splice modulation such as exon-skipping, although their therapeutic effect may be compromised by insufficient delivery. To address this issue, we investigated the synthesis of a 20-mer 2′-OMe PS oligonucleotide conjugated at 3′-end with ursodeoxycholic acid (UDCA) involved in the targeting of human DMD exon 51, by exploiting both a pre-synthetic and a solution phase approach. The two approaches have been compared. Both strategies successfully provided the desired ASO 51 3′-UDC in good yield and purity. It should be pointed out that the pre-synthetic approach insured better yields and proved to be more cost-effective. The exon skipping efficiency of the conjugated oligonucleotide was evaluated in myogenic cell lines and compared to that of unconjugated one: a better performance was determined for ASO 51 3′-UDC with an average 9.5-fold increase with respect to ASO 51. In the part of experimental materials, we found many familiar compounds, such as 6-Aminohexan-1-ol(cas: 4048-33-3Computed Properties of C6H15NO)
6-Aminohexan-1-ol(cas: 4048-33-3) may be used along with glutaric acid to generate poly(ester amide)s with excellent film- and fiber forming properties. It can undergo cyclization over copper supported on γ-alumina and magnesia to form hexahydro-1H-azepine.Computed Properties of C6H15NO
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