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Referencia número: HY-132611A

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Golodirsen sodium

(Synonyms: SRP-4053 sodium)

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Based on 1 Customer Validation

Golodirsen (SRP-4053) sodium is an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO). Golodirsen sodium restores the reading frame of the Duchenne muscular dystrophy (DMD) gene by modifying the splicing process of the pre-mRNA, skipping exon 53. Golodirsen sodium can restore the expression of the anti-myostatin protein. Golodirsen sodium can be used for the research of duchenne muscular dystrophy (DMD).

Para uso exclusivo en investigación. No vendemos a pacientes.

Pureza: 92.00%
Fòrmula: C₃₀₅H₄₈₁N₁₃₈Na₂₅O₁₁₂P₂₅
Peso molecular:8647 (free acid)

Almacenamiento:

-20°C, stored under nitrogen, away from moisture

* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)

Golodirsen sodium Chemical Structure

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Use of MCE Products

Fluorescent Dye

Strong Fluorescence Signal / Multi-Disciplinary Applications / High Fluorescence Specificity

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- In Vitro
- In Vivo
- Ensayo clínico
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Bioz

Actividad biológica

Descripciòn

Ver todos los productos específicos de isoformas DNA/RNA Synthesis :

In Vitro

Golodirsen sodium demonstrates efficient and specific exon 53 skipping ability in human fibroblasts, and is able to significantly restore the imbalance of DMD transcripts^[1].
Golodirsen sodium restores the expression of the anti-myostatin protein in human fibroblasts, but there are significant individual differences in the level of protein recovery, and it has no correlation with the jumping efficiency^[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

In Vivo

Golodirsen sodium (12-960 mg/kg (mice), 100-900 mg/kg (rats), 5-320 mg/kg (cynomolgus monkeys), i.v., single dose) exhibits consistent pharmacokinetic characteristics in multiple species (plasma half-lives: 2.1-8.7 h) and extensive biodistribution is observed in the target skeletal muscle tissues and the kidney^[2].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Ensayo clínico

NCT Number	Sponsor	Condition	Start Date	Phase A549
NCT01329991	Plexxikon\|		2011-05	PHASE1

Chemical Information

Appearance Solid
Peso molecular 8647 (free acid)
Fòrmula C₃₀₅H₄₈₁N₁₃₈Na₂₅O₁₁₂P₂₅
Color White to off-white
SMILES

[Golodirsen (sodium)]
Synonyms

SRP-4053 sodium
Envío

Room temperature in continental US; may vary elsewhere.
Almacenamiento

-20°C, stored under nitrogen, away from moisture

* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)

Pureza y Documentación

Select Batch:

Pureza: 99.98%

Assay: 99.98%

ee.: 99.98%

Ficha de datos (266 KB)
SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Korean - KR (252 KB)
- Portuguese - PT (252 KB)
COA (2659 KB)
Elemental Analysis Report (2659 KB)
HNMR (2659 KB)
CNMR (2659 KB)
RP-HPLC (2659 KB)
NP-HPLC (2659 KB)
MS (2659 KB)
LCMS (2659 KB)
IR (2659 KB)
GC (2659 KB)
OR (2659 KB)
SDS-PAGE (2659 KB)
SEC-HPLC (2659 KB)
Other Report (2659 KB)
Instrucciones de manejo (2242 KB)

Referencias

[1]. Rossi R, et al. Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells. Skelet Muscle. 2024 Nov 29;14(1):28. [Content Brief]

[2]. Goey, Andrew KL, et al. Characterization of Nonclinical Drug Metabolism and Pharmacokinetic Properties of Phosphorodiamidate Morpholino Oligonucleotides, a Novel Drug Class for Duchenne Muscular Dystrophy. Drug Metabolism and Disposition 52.12 (2024): 1396-1406.

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Calculadora de dilución

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Help & FAQs

How should lyophilized recombinant proteins be reconstituted and stored?

1. Before opening the cap, centrifuge the vial at 13000 rpm for 20-30 seconds. This step will ensure that any lyophilized powder that may have adhered to the cap or walls is collected at the bottom of the vial, minimizing the risk of product loss.

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MOQ

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100 mg

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Golodirsen sodium

Ver todos los productos específicos de isoformas DNA/RNA Synthesis

Actividad biológica

Chemical Information

Pureza y Documentación

Referencias

Calculators

Help & FAQs

Golodirsen sodium Related Classifications

HY-132611A Related Classifications