Morpholino oligonucleotide-mediated exon skipping for DMD treatment: Past insights, present challenges and future perspectives
Duchenne muscular dystrophy (DMD) is an X-linked genetic disease primarily affecting boys
causing loss of the dystrophin protein, ultimately leading to muscle wastage and death by …
causing loss of the dystrophin protein, ultimately leading to muscle wastage and death by …
Synthesis and biophysical studies of high-affinity morpholino oligomers containing G-clamp analogs
Successful syntheses of chlorophosphoramidate morpholino monomers containing tricyclic
cytosine analogs phenoxazine, G-clamp, and G8AE-clamp were accomplished. These …
cytosine analogs phenoxazine, G-clamp, and G8AE-clamp were accomplished. These …
Synthesis and Biophysical Properties of Triazole-Incorporated PMOs (TzPMOs): A Convergent, Click Ligation Approach
The synthesis of phosphorodiamidate morpholino oligonucleotides (PMOs) incorporating
single or double triazole rings in the backbone has been achieved via Cu (I)-catalyzed azide …
single or double triazole rings in the backbone has been achieved via Cu (I)-catalyzed azide …
Synthesis of Self Permeable Antisense PMO Using C5-Guanidino-Functionalized Pyrimidines at the 5′-End Enables Sox2 Downregulation in Triple Negative Breast …
Delivery of macromolecular drugs inside cells has been a huge challenge in the field of
oligonucleotide therapeutics for the past few decades. Earliest natural inspirations included …
oligonucleotide therapeutics for the past few decades. Earliest natural inspirations included …
Morpholine Scaffolds' Preparation for Foldamers' Design and Construction
N Schiaroli, C Loro, S Colombo… - Asian Journal of …, 2024 - Wiley Online Library
This review highlights the advances in the synthesis of morpholine building blocks useful for
artificial oligomers synthesis and peptide foldamers or biopolymers. Both morpholino …
artificial oligomers synthesis and peptide foldamers or biopolymers. Both morpholino …
N3-Methyluridine and 2′-O-Alkyl/2′-Fluoro-N3-methyluridine functionalized nucleic acids improve nuclease resistance while maintaining duplex geometry
Herein, we report the synthesis of 2′-O-alkyl/2′-fluoro-N 3-methyluridine (2′-O-alkyl/2′-
Fm 3 U) phosphoramidites and their incorporation in DNA and RNA oligonucleotides. The …
Fm 3 U) phosphoramidites and their incorporation in DNA and RNA oligonucleotides. The …
A convergent click ligation approach to the synthesis of triazole-incorporated PMOs and evaluation of hybridization properties
The synthesis of Phosphorodiamidate Morpholino Oligonucleotides (PMOs) incorporating
single triazole rings in the backbone has been achieved via Cu (I) catalyzed Azide-Alkyne …
single triazole rings in the backbone has been achieved via Cu (I) catalyzed Azide-Alkyne …
Synthesis and Biophysical Studies of High Affinity Morpholino Oligomers Containing G-clamp Analogs
Synthesis of chlorophosphoramidate morpholino monomers containing tricyclic cytosine
analogs phenoxazine, G-clamp, and G8AE-clamp were achieved and incorporated into 12 …
analogs phenoxazine, G-clamp, and G8AE-clamp were achieved and incorporated into 12 …