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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Explore more from The Transmitter
The genome offers a recipe, not a blueprint, for the brain
During brain development, genes set the rules and turn the knobs; self-organization does much of the rest.
The genome offers a recipe, not a blueprint, for the brain
During brain development, genes set the rules and turn the knobs; self-organization does much of the rest.
Autism research involving monkeys contends with uphill battle
Macaques and marmosets recapitulate the condition’s core traits better than mice can, but funding uncertainty and challenges at primate centers in the United States could make this work difficult to continue.
Autism research involving monkeys contends with uphill battle
Macaques and marmosets recapitulate the condition’s core traits better than mice can, but funding uncertainty and challenges at primate centers in the United States could make this work difficult to continue.
When is self-organization a mechanism in brain development?
Retinal waves offer a powerful example of a self-organizing system that can build structure without external guidance. The field would do well to embrace such phenomena more broadly as a type of mechanistic explanation.
When is self-organization a mechanism in brain development?
Retinal waves offer a powerful example of a self-organizing system that can build structure without external guidance. The field would do well to embrace such phenomena more broadly as a type of mechanistic explanation.