Rett syndrome
Recent articles
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.
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.
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.
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.
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.
FDA approval of trofinetide may spur further drug development for Rett
The drug, welcomed by patients, might be just the first of many.
FDA approval of trofinetide may spur further drug development for Rett
The drug, welcomed by patients, might be just the first of many.
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.
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.
Explore more from The Transmitter
Eli Sennesh talks about bridging predictive coding and NeuroAI
Predictive coding is an enticing theory of brain function. Building on decades of models and experimental work, Eli Sennesh proposes a biologically plausible way our brain might implement it.
Eli Sennesh talks about bridging predictive coding and NeuroAI
Predictive coding is an enticing theory of brain function. Building on decades of models and experimental work, Eli Sennesh proposes a biologically plausible way our brain might implement it.
In your New Year’s resolutions for 2025, consider public outreach
If every person in the neuroscience community committed to doing one thing, imagine the cumulative difference it would make.
In your New Year’s resolutions for 2025, consider public outreach
If every person in the neuroscience community committed to doing one thing, imagine the cumulative difference it would make.
Cerebellar SHANK3; telehealth coaching for caregivers; psychedelics
Here is a roundup of autism-related news and research spotted around the web for the last two weeks of December.
Cerebellar SHANK3; telehealth coaching for caregivers; psychedelics
Here is a roundup of autism-related news and research spotted around the web for the last two weeks of December.