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Spectrum: Autism Research News

Tag: gene therapy

April 2023
Long-tailed macaque in the canopy of a fruiting strangler fig tree, surrounded by ripening figs. Gunung Palung National Park, Borneo.

RNA therapy restores gene function in monkeys modeling Angelman syndrome

by  /  7 April 2023

The result raises hopes for an ongoing clinical trial in people — and offers fresh insight into the biology of imprinting and the UBE3A antisense transcript.

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February 2023
Illustration of a sheet of red and white pills, with the red pills arranged in the form of a question mark.

Going on Trial: Orphan drugs; CBD; bumetanide

by  /  28 February 2023

This month’s newsletter takes a close look at the orphan drug program in the United States, several cannabis-based therapies and a secondary analysis of bumetanide, among other new developments in autism-related drug trials.

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Research image of cell bodies of rett-like neurons, some of which were treated with epigenome editors.

Epigenome edits unmute MECP2 in Rett-like neurons

by  /  16 February 2023

The approach removes methyl tags from the gene and shields it from other silencing factors without changing the gene itself, raising hopes for a new treatment.

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Picture of a caged mouse with an EEG implanted.

Adult Angelman mice get some benefit from boosting UBE3A gene expression

by  /  9 February 2023

The treatment eases the animals’ sleep troubles, suggesting it has clinically meaningful effects beyond what was thought to be a critical window in early life.

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January 2023
Image of neurons during a seizure.

Calming neurons to quell seizures: Q&A with Gabriele Lignani and Dimitri Kullmann

by  /  3 January 2023

A new gene therapy approach for epilepsy tamps down neural activity on demand.

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December 2022
An illustration of doctors examining a larger-than-life DNA strand

Whole-genome trove ties new genes, variants to autism

by  /  12 December 2022

A massive update to the MSSNG dataset gives qualified researchers ready access to explore autism’s genetic architecture on a cloud-based platform.

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October 2022

New RNA tool prompts protein production in select cells

by  /  28 October 2022

The technique could be used to identify and control cells involved in autism.

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Jeremy Levin, chief executive officer of Ovid Therapeutics, stands against a bench in a lab.

What next for Angelman?

by  /  20 October 2022

A meeting in Texas reckons with the future of treatment, following two setbacks in 2020.

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September 2022
Set of 6 scans shows mice recovering MECP2 protein in their brainstem after being treated with RNA-editing viruses.

RNA rewrite improves breathing, survival in Rett mice

by  /  22 September 2022

The technique involves editing the cellular instructions to make MECP2 protein and partially restores its levels in the brainstem.

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August 2022

Promising preclinical results prompt Angelman therapy trial

by  /  25 August 2022

Roche’s gene therapy drug Rugonersen boosts expression of the protein missing in the syndrome in mice and monkeys, but whether it works in people remains to be seen.

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