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Spectrum: Autism Research News

Tag: gene therapy

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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September 2021

New method uses virus-like protein to package, deliver RNA

by  /  9 September 2021

A novel gene delivery system taps a protein found in people to encapsulate messenger RNA and transport it into cells.

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June 2021
lab technician pipetting in dramatic light

Alternative gene-therapy approaches take aim at Rett syndrome

by  /  16 June 2021

Methods that selectively increase levels of the Rett protein make for safer and more effective treatment strategies, some researchers say.

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CRISPR tool creates long-lasting epigenome alterations

by  /  11 June 2021

A new spin on the classic CRISPR gene-editing tool can alter the genome’s chemical tags and suppress gene expression for months.

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January 2021

Method may improve safety of gene therapies targeting the brain

by  /  28 January 2021

A new approach for delivering gene therapy to the brain reduces nerve damage in primates and could help make gene therapies for conditions related to autism safer.

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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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New gene therapy methods deliver promise

by  /  14 January 2021

Two unpublished studies detail improved techniques for delivering gene therapies to the brain.

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December 2020
researcher facing off with the coronavirus.

Rewind: Spectrum’s best from 2020

23 December 2020

Our staff picks the stories, podcasts and special reports that stood out from the rest this past year.

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

Hot topics in autism research, 2020

23 December 2020

The Spectrum team highlights five topics that distinguished autism research in 2020: diversity in data, gene therapies, subtyping, social circuitry and the ‘autism gene’ debate.

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November 2020
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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