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Tag: gene therapy

June 2021
lab technician pipetting in dramatic light
News

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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News / Toolbox

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
News / Toolbox

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.
News

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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News

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Two unpublished studies detail improved techniques for delivering gene therapies to the brain.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.
Features / Special Reports / Hindsight is 2020: The year in review

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
Features / Special Reports / Hindsight is 2020: The year in review

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
News

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.
Features / Deep Dive

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
News

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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