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Golodirsen

Name: Golodirsen
Brand: TargetMol
SKU: T38860
Rating: 4.5 (1 reviews)

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Catalog No. T38860Cas No. 1422959-91-8

Alias SRP-4053, Golodirsen

Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind to exon 53 of dystrophin pre-mRNA, used in the investigation of Duchenne muscular dystrophy (DMD).

Golodirsen

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Catalog No. T38860Alias SRP-4053, GolodirsenCas No. 1422959-91-8

Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind to exon 53 of dystrophin pre-mRNA, used in the investigation of Duchenne muscular dystrophy (DMD).

Pack Size	Price	USA Warehouse	Global Warehouse	Quantity
1 mg	$391	2-4 weeks	2-4 weeks
5 mg	$978	2-4 weeks	2-4 weeks

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In Stock Estimated shipping dateUSA Warehouse[1-2 days] Global Warehouse[5-7 days]

With extensive experience in compound synthesis, we can provide rapid custom synthesis services for this product according to your research needs.All TargetMol products are for research purposes only and cannot be used for human consumption. We do not provide products or services to individuals. Please comply with the intended use and do not use TargetMol products for any other purpose.

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Product Introduction

Bioactivity

Description	Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind to exon 53 of dystrophin pre-mRNA, used in the investigation of Duchenne muscular dystrophy (DMD).
In vitro	Golodirsen acts selectively on exon 53 of dystrophin pre-mRNA to cause its exclusion from the final mRNA sequence. This exon skipping restores the mRNA reading frame, facilitating the creation of a truncated yet functional dystrophin protein, which retains intact C and N-terminal regions[2].
Synonyms	SRP-4053, Golodirsen

Chemical Properties

Cas No.

1422959-91-8

Storage & Solubility Information

Storage

Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature.

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Please enter your animal experiment information in the following box and click Calculate to obtain the mother liquor preparation method and in vivo formula preparation method:

For example, your dosage is 10 mg/kg Each animal weighs 20 g, and the dosage volume is 100 μL . TargetMol | Animal experiments

A total of 10 animals were administered, and the formula you used is 5% TargetMol | reagent

DMSO+30% PEG300+5% Tween 80+60% Saline /PBS/ddH₂O. So your working solution concentration is 2 mg/mL。

Mother liquor preparation method: 2 mg of drug dissolved in 50 μL DMSO TargetMol | reagent

(mother liquor concentration of 40 mg/mL), if you need to configure a concentration that exceeds the solubility of the product, please contact us first.

Preparation method for in vivo formula: Take 50 μL DMSO TargetMol | reagent

main solution, add 300 μLPEG300 TargetMol | reagent

mix well and clarify, then add 50 more μL Tween 80, mix well and clarify, then add 600 more μLSaline /PBS/ddH₂O TargetMol | reagent

mix well and clarify

For Reference Only. Please develop an appropriate dissolution method based on your laboratory animals and route of administration.

All types of co-solvents required for the protocol, such asDMSO, PEG300/ PEG400, Tween 80, SBE-β-CD, corn oil are available for purchase on the TargetMol website with a simple click.

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Keywords

SRP4053SRP 4053