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Tag: Angelman syndrome

November 2020
Micrograph of nerve cells being targeted by CRISPR enzyme to activate the silenced gene in Angelman syndrome
News

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
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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April 2020
Illustration shows a boy with converging patterns overlapping; these lines signify autism and intellectual disability.
Features / Deep Dive

The blurred line between autism and intellectual disability

by  /  15 April 2020

Doctors often conflate autism and intellectual disability, and no wonder: The biological distinction between them is murky. Scientific progress depends on knowing where the conditions intersect — and part ways.

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January 2020
neurons firing in black and white
News

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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October 2019
UBE3A
News

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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Opinion / Viewpoint

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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August 2019
News

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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June 2019
children standing in a line, oldest to youngest
News

Timing of autism treatments may be key to their success

by  /  20 June 2019

The timing of treatment is crucial for conditions related to autism — and more so for some conditions than for others.

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April 2019
Human fetus with molecules going into it's brain
News

Injecting CRISPR into fetal brain may correct autism mutations

by  /  24 April 2019

Researchers are edging closer to a therapy for Angelman syndrome — a condition related to autism — that involves injecting the gene-editing enzyme CRISPR into the fetal brain.

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