Applied Evidence
Narrative ReviewDevelopmental medicine and child neurology2021

UBE3A reinstatement as a disease-modifying therapy for Angelman syndrome.

Ype Elgersma, Monica Sonzogni

PMID 33543479

WHAT IT FOUND

There are no original clinical results to act on.

This review covers UBE3A loss in Angelman syndrome and possible ways to restore UBE3A.

What this paper is

This is a review about UBE3A loss in Angelman syndrome and possible ways to restore UBE3A. It discusses gene therapy and antisense oligonucleotides. It does not report original methods or patient outcomes, so there is no finding here to change practice.

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The study

Certainty of evidence
Low

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    Cite

    Ype Elgersma, Monica Sonzogni UBE3A reinstatement as a disease-modifying therapy for Angelman syndrome. Developmental medicine and child neurology. 2021.

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