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Chimeric AAV vectors enabling antibody evasion and redosing for cardiac gene therapy

Chimeric AAV vectors enabling antibody evasion and redosing for cardiac gene therapy

Unmet Need Gene therapy using adeno-associated virus (AAV) vectors is severely limited by pre-existing immunity in 30–80% of adults and by the development of neutralizing antibodies after initial treatment, preventing redosing. There is a critical…

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Muscle-targeted gene therapy for SLC25A4 mitochondrial deficiency

Muscle-targeted gene therapy for SLC25A4 mitochondrial deficiency

Unmet Need There is a significant unmet need for disease-modifying therapies that address mitochondrial dysfunction in inherited cardiomyopathies and myopathies such as SLC25A4 deficiency. Existing treatments are limited to supportive care and heart transplantation, with…

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Novel AAV capsid variants for CNS-targeted gene therapies

Novel AAV capsid variants for CNS-targeted gene therapies

Unmet Need The development of effective gene therapies for central nervous system (CNS) diseases faces a critical bottleneck: current adeno-associated virus (AAV) vectors lack sufficient transduction efficiency and specificity for human brain cells. Standard AAV…

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Microbial Growth Foundational Model

Microbial Growth Foundational Model

Unmet Need Microbial growth dynamics provide crucial insights into microbial populations, with applications in drug discovery, personalized medicine, and synthetic biology. Advances in the field have resulted in an ever-growing list of known microbe species…

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Precision Engineering of Neural Circuits via Designer Connexins for Long-Term Cellular Integration

Precision Engineering of Neural Circuits via Designer Connexins for Long-Term Cellular Integration

Unmet Need Gap junctions are crucial for enabling the direct flow of ions and small molecules between cells, playing a significant role in synchronizing electrical activity in organs like the heart and brain. However, mutations…

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Morphological multiparameter filtration and persistent homology in mitochondrial image analysis

Morphological multiparameter filtration and persistent homology in mitochondrial image analysis

Unmet Need Current mitochondrial network analysis tools require significant manual intervention, making them labor-intensive, subjective, and prone to variability. These limitations hinder the reproducibility and accuracy needed for studying complex mitochondrial morphologies in disease diagnostics,…

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3D cavitation mapping to improve laser lithotripsy for the treatment of kidney stones

3D cavitation mapping to improve laser lithotripsy for the treatment of kidney stones

Unmet Need The prevalence of kidney stones is on the rise, having increased from approximately 4% in the 1970s to nearly 10% today. Approximately 1 in 10 of Americans will experience a kidney stone in…

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Acoustofluidic technologies for precision control in microfluidic systems

Acoustofluidic technologies for precision control in microfluidic systems

Unmet Need Achieving precise manipulation of particles, cells, and droplets in microscale liquid systems is a major challenge in life sciences, material sciences, and chemistry. Traditional microfluidic systems, which use hydrophobic channels and pressure differentials…

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A fluorescent probe for monitoring beta-secretase activity in cells

A fluorescent probe for monitoring beta-secretase activity in cells

Unmet Need Alzheimer’s Disease (AD) is characterized by the accumulation of amyloid-beta (Aβ) plaques, which lead to neurotoxicity, synaptic damage, and neuronal cell death. Current therapeutic approaches face significant challenges due to the complex nature…

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Novel Method for Creating and Modifying Stable Cell Lines for Enhanced Proteomic Analysis

Novel Method for Creating and Modifying Stable Cell Lines for Enhanced Proteomic Analysis

Unmet Need Characterizing the composition and function of protein complexes can facilitate the understanding of key cellular processes. Indeed, the global proteomics market was estimated to be USD 25.9 billion in 2021. However, the analysis…

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MEAPS: A method to discover bioactive compounds generated by gut microbes

MEAPS: A method to discover bioactive compounds generated by gut microbes

Unmet Need Microbial communities play important roles in environmental, animal, and human health. In humans, beneficial bacteria living within the intestinal tract can promote gut development, balance immunity, and enhance nutrient metabolism. Alternatively, colonization with…

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Anti-LYPD3 CAR T-cell therapy for the treatment of squamous cell carcinoma

Anti-LYPD3 CAR T-cell therapy for the treatment of squamous cell carcinoma

Unmet Need Chimeric Antigen Receptor (CAR) T-cell therapy, a form of immunotherapy, has proven highly effective against blood cancers such as leukemias and lymphomas. This therapy uses T-cells that are genetically engineering ex vivo to…

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