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Spectrum: Autism Research News

Tag: UBE3A

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

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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October 2019
UBE3A

Rat model mimics communication problems in Angelman syndrome

by  /  23 October 2019

Rats missing UBE3A, the gene mutated in people with Angelman syndrome, squeak frequently but tend not to be responsive to the play and squeaks of other rats.

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August 2019

What matters for protein linked to autism? Location, location, location

by  /  14 August 2019

One form of the protein implicated in Angelman syndrome and autism clusters in the nucleus, and it’s this form that may be critical to brain development.

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July 2019

Gene linked to multiple forms of autism may boost risk of death from seizures

by  /  18 July 2019

Mice with extra copies of UBE3A, a gene linked to autism and related conditions, are susceptible to death from seizures.

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