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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…
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…
Method for increasing gene expression in mammalian cells from transfected plasmids
Unmet Need Mammalian cell-based protein expression systems enable the production of mammalian proteins in the most native structure and activity. Thus, they have been commonly used for producing antibodies and therapeutic proteins, and more recently,…
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…