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Apazunersen

Angelman syndrome

Apazunersen is an antisense oligonucleotide (ASO) designed to target and inhibit the expression of the UBE3A antisense transcript (UBE3A-AS). This mechanism prevents the silencing of the paternally inherited allele of the UBE3A gene, facilitating the reactivation of the deficient protein. Apazunersen holds potential for research applications related to Angelman syndrome, offering insights into genetic regulation and therapeutic strategies for this neurodevelopmental disorder.

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Specifications

Manufacturer	Adooq Bioscience
CAS Number	2919324-01-7
Applications	Angelman syndrome
Formula	C₁₈₄H₂₂₄N₆₇O₉₆P₁₇S₁₇
Molecular Weight	5981.79
Synonyms	GTX-102

Storage & Solubility

In Vitro Solubility

Reported Solubility

No reported solubility data are available for this product.

Preparing Stock Solutions

Stock preparation is unavailable for this product molecular weight.

Calculated stock concentrations do not establish solvent solubility. Refer to the reported solubility data for experimental guidance.

Storage

Store powder at -20°C, keep desiccated.
In powder form, the chemical is stable for 36 months.
In solution, store at -20°C and use within 1 month to prevent loss of potency. Aliquot to avoid multiple freeze/thaw cycles.