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Antisense Therapy Restores Fragile X Protein Production in Human Cells

Antisense Therapy Restores Fragile X Protein Production in Human Cells
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Jonathank wattsUmass chanJoeld richterHuseyin mehmetSneha shahMichael tranfagliaElizabeth berry kravisArthurf koskinasUmass chan medical schoolUniversity medical centerProceedings of the national academy sciencesNational institutes of healthResearch foundationSimons foundation for autism researchBusiness developmentChan medical school

Multi-lab study hints at benefits of long-tested autism drug

The results lend support for clinical trials of arbaclofen in people with an autism-linked condition, the researchers say.

United statesRush university medical centerElizabeth berry kravisMassachusetts institute of technologyClinical research associatesSimons foundationSimons foundation autism research initiativeBrigitta gundersenSimons foundation autism researchMark bearMassachusetts institute

Company on brink takes psilocybin to trial for fragile X syndrome

The phase 2A trial has its detractors despite positive animal results and is being sponsored by a company that is struggling financially.

United statesUnited kingdomCity ofRush university medical centerCraig ericksonEmily osterweilMarvin hausmanElizabeth berry kravisViviana trezzaWilliam rascanEmma robinsonUniversity of edinburghHealth canadaUniversity of central floridaRoma tre universityUniversity of bristol

Are physicians ready to deploy future therapies?

As more gene therapies and complex therapies enter hospitals, physicians may not be well-equipped to administer these drugs.

Elizabeth berry kravisAllyson berent weisseFoundation for angelman syndrome therapeuticsRush universityWall street journalAngelman syndrome therapeuticsChicago based rush universityGene therapyHe wall street journalWall streetWall street journal

Adult Angelman mice get some benefit from boosting UBE3A gene expression | Spectrum

The treatment eases the animals’ sleep troubles, suggesting it has clinically meaningful effects beyond what was thought to be a critical window in early life.

District of columbiaUnited statesRush university medical centerBaylor college of medicineMingshan xueElizabeth berry kravisWu chenMichael sidorovNeuroscience research at children national hospitalCharles chen laboratory of mingshan xue brainCharles chen laboratory of mingshan xueBaylor collegeNeuroscience researchNational hospitalCharles chen