Rett syndrome
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.
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.
Supported by a $40 million NIH grant, Yale brain shuttle technology raises questions
Yale University claims its STEP platform might be able to deliver gene-editing tools into the brain via multiple routes. Researchers are eager to see more.
Supported by a $40 million NIH grant, Yale brain shuttle technology raises questions
Yale University claims its STEP platform might be able to deliver gene-editing tools into the brain via multiple routes. Researchers are eager to see more.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Explore more from The Transmitter
University probe leads to retraction of four neural tube studies
The University of Maryland conducted an internal investigation that identified image duplications and other data integrity issues in the studies, according to the retraction notices.
University probe leads to retraction of four neural tube studies
The University of Maryland conducted an internal investigation that identified image duplications and other data integrity issues in the studies, according to the retraction notices.
Highly cited autism paper earns expression of concern over data duplications
The notice, published in Nature last week, also addressed an image duplication identified by a prominent research-integrity sleuth.
Highly cited autism paper earns expression of concern over data duplications
The notice, published in Nature last week, also addressed an image duplication identified by a prominent research-integrity sleuth.
Govern neurodata by how it’s used, not where it’s from
New neurodata laws will shape how researchers gather, share and reuse brain data. A use-based governance protects basic researchers and those whose data are being used, without freezing science.
Govern neurodata by how it’s used, not where it’s from
New neurodata laws will shape how researchers gather, share and reuse brain data. A use-based governance protects basic researchers and those whose data are being used, without freezing science.