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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…
Rationally designed chimeric AAV capsids for enhanced immune evasion, cellular uptake, and transduction efficiency
Unmet Need Adeno-associated virus (AAV) vectors are a leading platform for in vivo gene therapy, yet their clinical potential is limited by key biological barriers. Pre-existing immunity to common AAV serotypes diminishes vector efficacy and…
RNA switch enabling stress-responsive regulation of protein expression for safer, more consistent gene and mRNA therapies
Unmet Need Current gene and mRNA therapies lack mechanisms to regulate protein expression in response to intracellular stress. Overexpression of therapeutic proteins can induce ER stress leading to unfolded proteins that do more harm than…
A novel all-in-one AAV system for efficient epigenome editing in neurodegenerative diseases
Unmet Need Neurodegenerative diseases such as Alzheimer’s and Parkinson’s affect over 55 million people worldwide, with prevalence expected to double by 2050 due to aging populations. These conditions are progressive, debilitating, and currently have no…
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…
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…
Methods and compositions to sense m6A RNA modifications using a genetically encoded sensor
Unmet Need Gene expression is highly regulated through the addition of chemical modifications to RNA molecules. One such modification is methylation of adenosine residues to form m6A, a modified nucleotide which is found in thousands…
Methods and compositions for treating protein aggregation diseases
Unmet Need Neurodegenerative disease affects more than 50 million people per year. Certain neurodegenerative diseases, like Alzheimer’s disease, result from the accumulation of amyloid-forming proteins in the brain, which results in cognitive impairment and has…
CRISPR-based antiviral treatment of coronavirus infections
Unmet Need The global market for antiviral drugs is a large one, topping $60B, and is getting larger due to the ongoing COVID-19 pandemic. Spurred on by a $3B investment from the U.S. government into…
A CRISPR-based system for modifying the length of 3’ untranslated regions (3’UTR) of an mRNA transcript that offers novel treatment strategies for prostate cancer and other diseases
Unmet Need Abnormal length changes in the 3’ untranslated region (3’UTR) of mRNAs are associated with many diseases such as systemic lupus erythematosus, α-thalassemia, facioscapulohumeral muscular dystrophy, and cancer. While existing technologies can manipulate expression…
A technique that promotes the stringent control of gene expression within specific cell types
Unmet Need A major challenge of gene therapy is to target only the intended cell- and/or tissue-type. For this reason, many gene therapies are delivered using adeno-associated virus (AAV) vectors with gene-specific promoters; however, off-target…