Award Data

Project Summary Sanfilippo disease (mucopolysaccharidosis type III; MPS III) is a devastating neurodegenerative lysosomal storage disorder of childhood whose pathologic features are neurologic: slowing of development, severe behavioral problems, progressive cognitive decline, dementia, and decline in motor skills leading to immobility, unresponsiveness, and death. We have focused on MPS IIID cause ...

Autism spectrum disorder (ASD) is a continuum of neurodevelopmental characteristics that includes deficits in communication and social interaction, as well as restrictive, repetitive interests and behaviors. ASD is an increasing public health concern, with about 1 in 45 American children diagnosed with ASD in 2014, a 10-fold increase in prevalence over the past 40 years. The effect of ASD on both ...

DESCRIPTION (provided by applicant): This fast-track SBIR will result in a new type of software system, called V-Motive, which will facilitate the combination of two proven autism behavioral interventions: Applied Behavior Analysis therapy (ABA) and Video Modeling (VM). V-Motive will have the potential to change how ABA therapy is performed by making it feasible for therapy providers to leverage V ...

Project SummaryThe purpose of the proposed work is first-in-man testing of novel painless defibrillation (PaDe) therapy. Sudden cardiac arrest (SCA) remains the number one killer in western civilization. The underlying mechanism of SCA is usually ventricular fibrillation (VF), and survival depends on prompt defibrillation. Existing defibrillators rely on the delivery of a brief high voltage shock ...

Project Summary/Abstract Inhibition of ceramide protects mice from death from the Radiation GI Syndrome (RGS). Administration of anti- ceramide single-chain Fv (scFv) 24 h post radiation exposure increases survival in intestinal stem cells and dramatically improves overall survival in mice. These data indicate that anti-ceramide scFv represents the first effective mitigator of lethal RGS. To advan ...

SUMMARY: RNA splicing plays a central role in the generation of proteome diversity and in gene regulation. Splicing impacts vital cellular processes, such as cell-fate and differentiation, acquisition of tissue-identity, and organ development. Thus, defects in splicing has been linked to many diseases, including spinal muscular atrophy, Duchenne muscular dystrophy, Parkinson’s disease, and sever ...

The goal of this proposal is to develop a commercial “plug and play”, user-friendly, powerful and reliable dynamic clamp system, to enable all neuronal electrophysiologists to be able to perform sophisticated dynamic clamp experiments, without any requirement for programming, engineering, or mathematical modeling skills. We will develop hardware and software specifically for neuroscience appli ...

Sympathetic innervation of the heart plays a critical role in arrhythmogenesis and recent studies have highlighted the role of surgical sympathetic denervation in management of refractory ventricular arrhythmiasThe procedure although safe is very invasive and performed via thoracoscopy using single lung ventilationThis is not tolerated by patients with lung disease or severe structural heart disea ...

Project SummaryThe expression of emotions through speech is an essential element of human communicationyet individuals with speech impairments who rely on speech generating devicesSGDsto talk are largely deprived of this communicative channelNo one should be forced to sound blaswhen in fact they re elated or to sound apathetic when they intend to scoldWhile certain expressive speech modes are offe ...

Project Summary Genetically-modified mouse models have proven to be essential for the production of antibody-related biological drugs (biologics). To date, the majority of biologics originate from mouse models, and small animal models are used not only to generate the antibodies, but also as a platform for further optimization and testing of the biologics. Unmet Need: Camelid-based antibodies, whi ...