Rett syndrome
Recent articles
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.
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.
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.
Explore more from The Transmitter
Leucovorin saga, and more
Here is a roundup of autism-related news and research spotted around the web for the week of 15 June.
Leucovorin saga, and more
Here is a roundup of autism-related news and research spotted around the web for the week of 15 June.
Models at the speed of thought: How AI coding is reshaping theoretical neuroscience
Agentic coding makes it possible to specify a neuroscience model in hours instead of months. Seven neuroscientists weigh in on what that tectonic change may bring to the field.
Models at the speed of thought: How AI coding is reshaping theoretical neuroscience
Agentic coding makes it possible to specify a neuroscience model in hours instead of months. Seven neuroscientists weigh in on what that tectonic change may bring to the field.
Writing science that humans and machines can read
Large language models are now routinely used to search, summarize and synthesize the literature at scales impossible for any individual researcher—yet scientific publishing has not adapted to that reality.
Writing science that humans and machines can read
Large language models are now routinely used to search, summarize and synthesize the literature at scales impossible for any individual researcher—yet scientific publishing has not adapted to that reality.