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Spectrum: Autism Research News

Tag: UBE3A

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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Photograph of lab researchers at Ultragenyx

Gene therapy trial for autism-linked condition is put on hold

by  /  5 November 2020

All five participants in a clinical trial of a gene therapy for Angelman syndrome experienced leg weakness, leading sponsors to pause the study.

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October 2020
Ralf Schmid, the research director in Neurodevelopmental diseases at the Gene Therapy Program Orphan Disease Center, loads replicated mouse dna into wells in the gel before applying electric current to test the viability of the dna.

A quest for Quincy: Gene therapies come of age for some forms of autism

by ,  /  14 October 2020

A gene therapy for Angelman syndrome stands at the forefront of efforts to treat autism-linked conditions that stem from single genes.

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January 2020
neurons firing in black and white

Brain organoids may shed light on seizures in Angelman syndrome

by  /  22 January 2020

The mutation that causes Angelman syndrome makes neurons hyperexcitable, which may explain the frequent seizures that most people with the syndrome have.

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December 2019

Five hot topics in autism research in 2019

by  /  23 December 2019

This year’s hot topics in autism research center around brain organoids, heart rate, the gut microbiome, treatment timing and early detection.

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