SUMMARY The CIRTS-4GT3 platform is a compact, programmable system that uses guide RNAs and human protein domains to boost production of specific proteins in cells, offering a safe and targeted therapy for genetic disorders caused by insufficient protein levels The Unmet Need:… Read More
SUMMARY Inrich-seq is a new method that selectively enriches and sequences inosine-containing DNA or RNA, enabling highly sensitive, genome-wide detection of off-target effects from adenine base editing in human cells The Unmet Need: Techniques need to rigorously evaluate and profile any unintended… Read More
SUMMARY The engineered hypercompact enAsCas12f system addresses CRISPR-Cas systems deliverability issues while offering enhanced gene-editing high specificity making it ideal for therapeutic applications using AAV delivery. The unmet Need: New CRISPR-systems with greater deliverability and higher gene editing capabilities CRISPR-Cas (clustered regularly interspaced short… Read More
SUMMARY The Unmet Need: Methods for efficient, reversible integration and excision of large DNA Genome engineering is a rapidly advancing field with immense potential for synthetic biology and gene therapy. The ability to precisely modify DNA sequences within living cells is crucial… Read More
SUMMARY Engineered TadA deaminase via directed evolution to produce CRISPR cytosine base editors that precisely convert target cytosines to thymines in specific DNA contexts with high efficiency and minimal off-target effects The Unmet Need: Gene editing tools that exclusively acting on cytosine… Read More
SUMMARY The field of metabolic disease treatment has long been challenged by the need for precise, long-term regulation of critical hormones and proteins that manage glucose homeostasis and energy balance. Current therapeutic strategies for conditions such as obesity and diabetes struggle to provide a sustained and adjustable intervention, leaving patients… Read More
SUMMARY Polymersomes are a type of nanoscale vesicle made from block copolymers and can encapsulate a variety of therapeutic agents including proteins and nucleic acids. These nanovesicles are assembled at room temperature and incorporate features such as permanently charged moieties for stability and mannosylation for targeted delivery. Read More
SUMMARY A breakthrough method to systematically select for optimal small guide RNAs using an unbiased approach for improved accuracy and efficiency of all systems of site-directed RNA editing. The Unmet Need: In vitro assays that reliably predict guide RNA efficiency and specificity before in… Read More
SUMMARY Groundbreaking advancement in the field of mRNA topical delivery systems, providing superior penetration, stability, and biocompatibility compared to existing solutions. It opens up new possibilities for both therapeutic and cosmetic applications, paving the way for more effective and user-friendly treatments. The unmet… Read More
SUMMARY A novel platform, bacterial deaminase-enabled recoding of RNA (DECOR), utilizing an engineered bacterial adenosine deaminase to achieve programmable adenosine-to-inosine (A-to-I) editing on single-stranded RNA. The Unmet Need: Novel effector proteins for programmed RNA editing RNA editing has emerged as a promising therapeutic strategy… Read More
SUMMARY Many human diseases arise from insufficient protein levels, often due to genetic mutations that result in haploinsufficiency or loss of protein function. Effectively treating these conditions necessitates therapeutic strategies capable of increasing the production of specific, vital proteins. There is a significant unmet need for programmable methods to… Read More
SUMMARY An understanding of protein-protein interactions (PPIs) is critical to uncovering new disease pathophysiology and for developing small molecule and antibody treatments. Problematically, current approaches to assay PPIs are limited by sensitivity, signal to noise ratio, and the need to carefully optimize the assay for each new application. The inventors… Read More