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Spectrum: Autism Research News

Tag: gene therapy

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Two unpublished studies detail improved techniques for delivering gene therapies to the brain.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

by  /  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

by  /  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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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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May 2020

Reversing SHANK3 mutations in mice mitigates autism-like traits

by  /  28 May 2020

Correcting an autism gene mutation in fetal mice lessens some autism-like behaviors after birth.

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November 2019
Chromosomes swapping DNA

CRISPR technique tracks genetic activity in real time

by  /  8 November 2019

A new method scans gene expression in live cells and could help scientists study autism genes.

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October 2019

Angelman syndrome’s silent gene points way forward for autism therapies

by  /  21 October 2019

Advances in research and help from families have brought scientists to the brink of an effective therapy for Angelman syndrome.

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December 2018
whimsical machine takes in various data and spits out an organized version on the other end

Notable papers in autism research in 2018

by  /  21 December 2018

This year’s list of top papers highlights new dimensions in our understanding of autism genetics and hints at novel treatments.

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