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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 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…
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 cDNA sequence encoding the ATX protein (Human ATX cDNA)
Value Proposition ATX is an active protein kinase that plays a role in cellular stress responses, and defects in ATX also play a role in the development of cancer and other proliferative disorders. Thus, small…