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Spectrum: Autism Research News

Tag: gene therapy

June 2022

Gene therapy targets interneurons to tackle Dravet syndrome

by  /  13 June 2022

The approach, tested in mice, selectively boosts the expression of the autism-linked gene SCN1A in a subgroup of inhibitory cells.

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Organoids test gene therapies for Pitt-Hopkins syndrome

by  /  2 June 2022

Strategies to replace or compensate for mutated copies of the TCF4 gene could lead to treatments for this profound form of autism, a new study suggests.

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May 2022
A woman in a white lab coat uses a micropipette

Modified CRISPR tool boosts UBE3A levels in mice

by  /  20 May 2022

A deactivated form of the gene editor restores UBE3A expression in mice and human neurons without cutting the genome. It may hold promise for future Angelman gene therapies.

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January 2022

Reimagining the newborn screening system: Q&A with Don Bailey

by  /  27 January 2022

The current system to approve genetic and metabolic conditions for newborn screening can’t keep pace with research on new therapies. Don Bailey wants to bring it up to speed.

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Neuron-specific virus overcomes barriers to brain-related gene therapy

by  /  14 January 2022

A new viral variant can deliver genes exclusively to the brain, overcoming a key hurdle in treating neurological conditions using gene therapy.

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November 2021

New gene-editing tools delete long stretches of DNA

by  /  17 November 2021

Two new methods make it possible to delete long sections of the genome, expanding the reach of CRISPR gene editing.

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Gene therapy for rare form of autism may treat another

by  /  11 November 2021

A MECP2 gene therapy for Rett syndrome eases repetitive behaviors, anxiety and hyperactivity in a mouse model of Pitt-Hopkins syndrome.

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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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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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