Rett syndrome

Recent articles

A mouse sits on a gloved hand.

Molecular changes after MECP2 loss may drive Rett syndrome traits

Knocking out the gene in adult mice triggered up- and down-regulated expression of myriad genes weeks before there were changes in neuronal function.

By Chloe Williams
20 March 2025 | 5 min read
Research image of frog cells.

Many autism-linked proteins influence hair-like cilia on human brain cells

The finding may help explain autism’s association with multiple co-occurring conditions that involve cilia defects.

By Charles Q. Choi
9 January 2025 | 4 min read
Photograph of a syringe.

What’s next for brain-directed gene therapy after death in Neurogene trial

The incident highlights that viral vectors can trigger deadly immune responses even when delivered directly to the nervous system.

By Calli McMurray
26 November 2024 | 6 min read
Research image of toxoplasma gondii showing cysts it creates as well as the MECP2 protein that it can deliver to neurons.

Parasite-based tool delivers MECP2 and other proteins to neurons

A method that uses a common brain parasite could help replenish the proteins deficient in Rett syndrome and other conditions.

By Giorgia Guglielmi
12 September 2024 | 4 min read
A digital rendering of a strand of RNA

New RNA editor boasts increased versatility, safety

The “PRECISE” technique reprograms cells in a way that, unlike DNA editors, avoids potentially permanent off-target effects.

By Charles Q. Choi
11 April 2024 | 4 min read
Illustration of a sheet of red and white pills, with the red pills arranged in the form of a question mark.

New template streamlines sharing clinical trial results

This month’s Going on Trial newsletter explores a new tool for communicating with clinical trial participants and their families, among other drug development news.

By Calli McMurray
31 October 2023 | 7 min read
Illustration of a sheet of red and white pills, with the red pills arranged in the form of a question mark.

Trials test utility of EEG biomarkers for autism-related conditions

This month’s Going on Trial newsletter dives into an electroencephalography biomarker that could track the efficacy of treatments for dup15q and Angelman syndromes, among other drug development news.

By Calli McMurray
31 August 2023 | 7 min read
A hand reaches from above to add a pill to a stack that is resting against the x-axis of a graph.

How organ-on-a-chip models can help drug development

This month’s Going on Trial newsletter explores how organ-on-a-chip models could smooth the transition from preclinical to clinical trials, among other drug development news.

By Calli McMurray
31 July 2023 | 5 min read
Illustration of a sheet of red and white pills, with the red pills arranged in the form of a question mark.

Going on Trial: Gene therapy for Rett; return to arbaclofen

This month’s newsletter looks at the early safety data from the first gene therapy trial for Rett syndrome, among other drug development news.

By Calli McMurray
29 June 2023 | 6 min read
Illustration of a room with DNA sequence wallpaper and three doors leading to abstract new environments.

The future of autism therapies: A conversation with Lilia Iakoucheva and Derek Hong

If a therapy for autism’s core traits makes it to market, it will likely take one of three forms, the researchers say.

By Peter Hess
14 April 2023 | 5 min read

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Gabriele Scheler reflects on the interplay between language, thought and AI

She discusses how verbal thought shapes cognition, why inner speech is foundational to human intelligence and what current artificial-intelligence models get wrong about language.

By Paul Middlebrooks
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Data streams into a transparent box.

Accepting “the bitter lesson” and embracing the brain’s complexity

To gain insight into complex neural data, we must move toward a data-driven regime, training large models on vast amounts of information. We asked nine experts on computational neuroscience and neural data analysis to weigh in.

By Eva Dyer, Blake Richards
26 March 2025 | 8 min read