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Spectrum: Autism Research News

Tag: UBE3A

February 2023
Picture of a caged mouse with an EEG implanted.

Adult Angelman mice get some benefit from boosting UBE3A gene expression

by  /  9 February 2023

The treatment eases the animals’ sleep troubles, suggesting it has clinically meaningful effects beyond what was thought to be a critical window in early life.

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January 2023
Jill Silverman sits at her desk with her dog.

Beyond the bench: Finding solitude with Jill Silverman

by  /  17 January 2023

The people-focused researcher shares her secrets to carving out time for grant writing, creating work-life balance and letting go of unread emails.

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August 2022

Promising preclinical results prompt Angelman therapy trial

by  /  25 August 2022

Roche’s gene therapy drug Rugonersen boosts expression of the protein missing in the syndrome in mice and monkeys, but whether it works in people remains to be seen.

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An Ultragenyx employee removes materials from a lab freezer.

Angelman therapy appears safer in restarted trial

by  /  5 August 2022

Interim results from the previously paused trial suggest that doses of the experimental gene therapy drug GTX-102 are well tolerated in children with the autism-linked condition.

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February 2022

Enrichment lessens Angelman-like traits in male mice

by  /  2 February 2022

Model animals don’t develop the usual behavioral and motor problems when reared in an enclosure containing exercise wheels, toys and treats.

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November 2021
UBE3A expression in mouse brain slices, shown in green.

One-shot gene therapy for Angelman syndrome shows promise in mice

by  /  29 November 2021

A potential new gene therapy delivered via a single injection mimics ratios of proteins normally found in cells.

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Dispatches from SfN 2021: Mitochondria, Rett therapy and oxytocin

These short reports from Spectrum journalists highlight some of the autism-related findings that caught our attention at the meeting this past week.

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October 2021
Medicine being drawn out of a vial with a syringe.

Paused Angelman syndrome trial to restart

by  /  7 October 2021

After imposing a pause in November 2020, the U.S. Food and Drug Administration has cleared the way for a clinical trial of a gene therapy for Angelman syndrome.

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January 2021
Illustration of firing neurons.

Autism protein may shape neuronal firing patterns

by  /  14 January 2021

Cells with excess UBE3A, an autism-linked protein, have atypical firing properties that can be corrected by limiting the protein’s levels, according to new research.

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November 2020
Micrograph of nerve cells being targeted by CRISPR enzyme to activate the silenced gene in Angelman syndrome

Prenatal CRISPR therapy blocks Angelman syndrome traits in mice

by  /  9 November 2020

A gene-editing treatment shows long-lasting effects in a mouse model of Angelman syndrome, a genetic condition related to autism.

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