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
‘Push-pull’ recipe for neural wiring used in multiple brain regions
A versatile pair of proteins steers neurons toward their targets and helps establish the brain’s sensory maps, new studies suggest.
‘Push-pull’ recipe for neural wiring used in multiple brain regions
A versatile pair of proteins steers neurons toward their targets and helps establish the brain’s sensory maps, new studies suggest.
Reward-learning algorithm hardwired into dopamine circuit
The finding bolsters the canonical model of reward prediction error, which has come under scrutiny in recent years.
Reward-learning algorithm hardwired into dopamine circuit
The finding bolsters the canonical model of reward prediction error, which has come under scrutiny in recent years.
Exclusive: Brain and spinal cord institute halts research, citing funding problems
The Burke Neurological Institute, which calls itself “the only research institute in the U.S. dedicated to finding treatments to repair the brain and spinal cord,” ceased research operations on 22 May.
Exclusive: Brain and spinal cord institute halts research, citing funding problems
The Burke Neurological Institute, which calls itself “the only research institute in the U.S. dedicated to finding treatments to repair the brain and spinal cord,” ceased research operations on 22 May.