MECP2

Recent articles

Finding the Rett syndrome gene and ways to fix it

Kevin Mitchell talks with Huda Zoghbi about her work identifying the MECP2 gene, its role in the brain and therapies to address its dysfunction.

By Kevin Mitchell
21 September 2026 | 4 min read
DNA strand.

Exon-skipping approach boosts levels of key Rett syndrome protein

Deleting a small region of the MECP2 gene partially restored function in neurons derived from people with Rett-associated variants.

By Giorgia Guglielmi
20 March 2026 | 5 min read
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 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

Autism-linked genes shape touch processing through different mechanisms, at different times

Whereas some mice display hypersensitivity to touch only as adults, others respond that way from birth.

By Sarah DeWeerdt
21 March 2024 | 5 min read
Research video of a zebrafish larva (zoomed in on the gut) being given glucose.

On the periphery: Thinking ‘outside the brain’ offers new ideas about autism

Neuronal alterations outside the brain may help to explain a host of the condition’s characteristic traits, including sensory changes, gut problems and motor differences.

By Sarah DeWeerdt
13 April 2023 | 20 min read
Drop of liquid medicine with air bubbles is dripping from a pipette on a light pink background.

FDA approval of trofinetide may spur further drug development for Rett

The drug, welcomed by patients, might be just the first of many.

By Shaena Montanari
16 March 2023 | 8 min read
Illustration of a gloved hand reaching down to place a pill bottle on a pedestal.

Debut drug for Rett syndrome at edge of approval

The U.S. Food and Drug Administration plans to make an approval decision on the first-ever drug for girls and women with Rett syndrome by 12 March.

By Shaena Montanari
2 March 2023 | 8 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: Orphan drugs; CBD; bumetanide

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.

By Peter Hess
28 February 2023 | 7 min read
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

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.

By Nora Bradford
16 February 2023 | 5 min read

Explore more from The Transmitter

Research image of perineuronal nets.

Organoids missing the TSC2 gene form rogue astrocytes; and more

Here is a roundup of autism-related news and research spotted around the web for the week of 28 September.

By Jill Adams
29 September 2026 | 2 min read
Research diagram of task-aligned activity rasters.

Cerebellar neurons reorient brain activity to differentiate similar behaviors

The findings provide a new perspective on how granule cells help to generalize learning across tasks.

By Holly Barker
29 September 2026 | 4 min read
Illustration of waterfall inside a woman's brain.

Revisiting the neural correlates of consciousness

Life happens in real time, but experience may be composed in ragtime—like the music genre, full of offbeat notes and uneven rhythms. The assumption that waking life consists of a “stream” of conscious representations has inadvertently led consciousness science to its current impasse.

By Francis T. Fallon, Michael Pitts
28 September 2026 | 7 min read