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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…
Viral-mediated humanized mouse model for Parkinson’s disease
Unmet Need Parkinson’s disease (PD) is a progressive neurodegenerative disorder characterized by the loss of dopaminergic neurons in the midbrain and the accumulation of α-synuclein aggregates (Lewy bodies), leading to motor and cognitive impairments. PD…
Novel TRPV4-Targeted Therapy for Arrhythmic Cardiomyopathy
Unmet Need Arrhythmic cardiomyopathy (ACM) is a life-threatening condition characterized by fatty-fibrotic remodeling of the heart muscle, leading to arrhythmias, heart failure, and sudden cardiac death. While 60% of ACM cases are linked to known…
Antigenome Platform: An autoantigen discovery pipeline
Unmet Need The human body contains approximately 60 trillion antibodies per microliter of blood in circulation, creating a vast and complex network of immune interactions. Identifying the targets of this plethora of antibodies is a…
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…
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,…
Novel epigenome editors for multiplexed gene regulation
Unmet Need Epigenome editors activate or inhibit gene expression without modifying the underlying DNA sequence. Consequently, these editors can reversibly fine-tune gene expression without the risk of permanently altering the genome or off-target editing. Epigenome…
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…
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…
Identification and application of novel lipoglycodepsipeptide antibiotics
Unmet Need Antibiotic resistance (ABR) is a global public health concern. In 2019, an estimated five million deaths were attributed to antibiotic resistant pathogens. A recent study estimates that ABR could cause nearly ten million…
Method for the in vitro differentiation of human dendritic cells from hematopoietic stem cells for research and immunotherapy development
Unmet Need Dendritic cells (DCs) play a critical role in the adaptive immune response by capturing and presenting antigens to activate cytotoxic T-cells. As such, naturally occurring DCs are thought to have crucial antitumor activity…
An organ chip model of the mammalian joint
Unmet Need Human movement relies on the function of cartilage-containing joints. In conditions such as osteoarthritis (OA), the cartilage within the joints becomes inflamed and degrades, leading to intense debilitating pain when bending joints. Despite…