INTRASTRIATAL ADMINISTRATION OF AAV5-MIHTT IN NON-HUMAN PRIMATES AND RATS IS WELL TOLERATED AND RESULTS IN MIHTT TRANSGENE EXPRESSION IN KEY AREAS OF HUNTINGTON DISEASE PATHOLOGY

Huntington disease (HD) is a fatal, neurodegenerative genetic disorder with aggregation of mutant Huntingtin protein (mutHTT) in the brain as a key pathological mechanism.There are currently no disease modifying therapies for HD; however, HTT-lowering therapies hold promise.Recombinant adeno-associated virus serotype 5 expressing a microRNA that ta

The distribution of woody biomass in savannas reflects spatial patterns fundamental to ecosystem processes, such as water flow, competition, and herbivory, and is a key contributor to savanna ecosystem services, such as fuelwood supply.While total precipitation sets an upper bound on savanna woody biomass, the extent to which substrate and terrain

The Pelagos Sanctuary hosts eight resident cetacean species.Genetically different from Atlantic populations, their conservation is essential to preserve the whole biodiversity of the Ligurian Sea.The NATURA 2000 network is currently identified as one of the most efficient conservation tools due to its legally binding nature; however, its effectiven

Wnt signaling is an important pathway in health and disease and a key scooters regulator of stem cell maintenance, differentiation, and proliferation.During heart development, Wnt signaling controls specification, proliferation and differentiation of cardiovascular cells.In this regard, the role of activated Wnt signaling in cardiogenesis is well d