SRP-5051 is the first investigational treatment using Sarepta's next-generation PPMO platform, which is designed around a proprietary cell-penetrating peptide conjugated to the phosphorodiamidate morpholino
oligomer (PMO) backbone with the goal of increasing drug concentration in muscle tissue.
It's a phosphodiamidate morpholino
oligomer engineered to treat DMD patients who have generic mutations, subject to skipping exon 53 of the dystrophin gene.
- Marketed drugs for DMD include the FDA-approved drugs Emflaza, a glucocorticoid with anti-inflammatory and immunosuppressant properties, and Exondys 51, a novel phosphorodiamidate morpholino
oligomer designed to induce the skipping of exon 51 in the dystrophin gene.
Sarepta announced that it has completed the submission of its rolling new drug application, or NDA, seeking accelerated approval for golodirsen, a phosphordiamidate morpholino
oligimer engineered to treat those patients with Duchenne muscular dystrophy who have genetic mutations subject to skipping exon 53 of the Duchenne gene.
We used a previously published sox9b morpholino
to determine whether sox9b expression regulates the expression of slincR.
The use of peptide-conjugated phosphorodiamate morpholino
oligomers could in future become a standardised practice in humans.
The company stated that it has received final minutes from a February 2018 Type C meeting held with the Division of Neurology Products, United States Food and Drug Administration (the Division), to solicit the Division's guidance on the development pathway for the company's therapeutic candidate, golodirsen, a phosphordiamidate morpholino
oligimer designed to treat those patients with Duchenne muscular dystrophy who have genetic mutations subject to skipping exon 53 of the DMD gene.
monolayers: Preparation and label-free DNA analysis by surface hybridization," Journal of the American Chemical Society, vol.
Noteworthy, knockdown models generated by the standard morpholino
method show discrepancy when compared to genome-editing based approach .
We injected morpholino
(MO), an antisense oligonucleotide sequence specifically targeting the transcribed mRNA, into zebrafish zygotes to block the translation of CRMP2 .
In order to purify, protein precipitated was dissolved in 20 mM morpholino
ethane sulfonic acid monohydrate buffer (pH = 5)and the sample was dialyzed using dialysis bag with 12000 MW pore in MES buffer to eliminate the waste materials (Bailey et al.
For double transgenic experiments microinjected with Cxcl8a and Cxcl8b morpholino
(MO), the neutrophils in circulation were quantified in the caudal vein using a 5 min movie with 10.5 s intervals in each embryo.