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Novel epigenome editors for multiplexed gene regulation

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…

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Method for increasing gene expression in mammalian cells from transfected plasmids

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,…

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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

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…

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A technique that promotes the stringent control of gene expression within specific cell types

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…

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