WebNov 19, 2024 · Phosphorodiamidate morpholino oligomer (PMO)-mediated exon skipping is a therapeutic approach that applies to many Duchenne muscular dystrophy (DMD) patients harboring out-of-frame deletion mutations in the DMD gene. In particular, PMOs for skipping exon 44 have been developing in clinical trials, such as the drug NS-089/NCNP-02. A Morpholino, also known as a Morpholino oligomer and as a phosphorodiamidate Morpholino oligomer (PMO), is a type of oligomer molecule (colloquially, an oligo) used in molecular biology to modify gene expression. Its molecular structure contains DNA bases attached to a backbone of … See more Morpholino oligos were conceived by Summerton (Gene Tools) at AntiVirals Inc. (now Sarepta Therapeutics) and originally developed in collaboration with Weller. See more Morpholinos do not trigger the degradation of their target RNA molecules, unlike many antisense structural types (e.g., See more For a Morpholino to be effective, it must be delivered past the cell membrane into the cytosol of a cell. Once in the cytosol, Morpholinos freely diffuse between the cytosol and nucleus, … See more • Wiley-Liss, Inc. Special Issue: Morpholino Gene Knockdowns of genesis Volume 30, Issue 3 Pages 89-200 (July 2001). This is a special issue of Genesis that consists of a series of peer-reviewed short papers using Morpholino knock downs of gene function in various … See more Morpholinos are synthetic molecules that are the product of a redesign of natural nucleic acid structure. Usually 25 bases in length, they bind to complementary sequences of RNA … See more Morpholinos have become a standard knockdown tool in animal embryonic systems, which have a broader range of gene expression than adult cells and can be strongly affected by … See more • Oligonucleotide synthesis • Nucleic acid analogue See more
Fully automated fast-flow synthesis of antisense
WebOct 4, 2024 · Structure and sequences of the model ASO constructs studied. A) Chemical structure of a phosphorodiamidate morpholino oligomer (PMO).B) The sequences for the single-stranded sense-NA, antisense-NA, antisense-PMO, duplex NA, and PMO:NA hybrid. The antisense-NA and antisense-PMO sequences are written 3´ to 5´ to show how they … WebIntroduction Duchenne muscular dystrophy (DMD) is a progressive disease that leads to damage of muscle and myocardium due to genetic abnormalities in the dystrophin gene. In utero cell... rch haguenau
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WebPeptides were conjugated to PMO through an amide linkage at the 3` end of the phosphorodiamidate morpholino oligonucleotide (PMO), followed by purification by high-performance liquid chromatography (HPLC) and analyzed by MALDI-TOF MS (for HPLC chromatogram and MALDI-TOF data, see Supplementary Figure S1). WebAn antisense phosphorodiamidate morpholino oligomer (PMO) complementary to the mRNA of the essential gene acpP (which encodes the acyl carrier protein required for lipid biosynthesis) in ... WebDec 7, 2024 · This is the first clinical data from SRP-5051, an investigational treatment that uses Sarepta’s peptide phosphorodiamidate morpholino oligomer (PPMO) technology. PPMO technology includes a proprietary cell-penetrating peptide that is conjugated to Sarepta’s PMO backbone with the goal of increasing cellular uptake of drug in the muscle … rch hay fever