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Brain of little angel donated for research after 3yo dies from rare Angelman Syndrome

Brain of little angel donated for research after 3yo dies from rare Angelman Syndrome
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Sunshine coastUnited statesNew south walesUnited kingdomMeagan crossHarry angelmanSaige snelgarEmma snelgarRyan snelgarBlair snelgarAngelman associationInternational angelman dayFoundation for angelman syndromeA uc davis healthFoundation for angelman syndrome therapeuticsFoundation for angelman syndrome therapeautics

FAST joins with University of Pennsylvania to develop investigational AAV gene therapy for Angelman syndrome

The Foundation for Angelman Syndrome Therapeutics (FAST) announced today that the non-profit organization has entered into an exclusive global collaborative research and development agreement with the University of Pennsylvania to develop an investigational adeno-associated virus (AAV) gene therapy for Angelman syndrome (AS).

Jim wilsonRoseh weissLily ramseyAllyson berentGene therapy programPerelman school of medicineDisease centerFoundation for angelman syndrome therapeuticsUniversity of pennsylvania pennUniversity of pennsylvaniaAngelman syndrome therapeuticsChief science officerOrphan disease centerPerelman schoolAngelman syndromeGene therapy

Couragene co-founders awarded $40M NIH grant

Couragene co-founders awarded $40M NIH grant
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United statesAmy liaoJianbing zhouJennifer panagouliasAllyson berentYong hui jiangElizabeth berry kravisTranslational researchPediatric neurosciencesf ast centerFoundation for angelman syndrome therapeuticsYale school of medicineNorth brunswick basedNational instituteSomatic cell genome editingYale schoolCouragene chief medical officer jiang

Large NIH Grant Supports CRISPR-based Gene Therapy Development for Brain Diseases

The two-phase grant will support research into a novel CRISPR-based gene-editing technology and delivery platform for targeting neurogenetic diseases.

United statesJulie wolfCaihong qiuKimberly greenbergAllyson berentYong hui jiangJames dziuraKathleen cardinaleHui zhangNigel bamfordJennifer panagouliasJiangbing zhouElizabeth berry kravisEric velazquezCaroline hendryMichele spencer manzon

NIH awards grant to Yale researchers for developing gene editing platform

The National Institute of Health announced a $40 million grant for researchers to develop a CRISPR-mediated platform for genome editing in the brain and to treat genetic disorders.

Tim taiJames mcpartlandYong hui jiangValentina simonJiangbing zhouAlana newhouseDrug administrationTimothy dwight collegeFoundation for angelman syndrome therapeuticsNational institutes of healthRush university medical centerYale school of medicineNational institutesYale schoolAngelman syndrome therapeuticsYale developmental disabilities clinic