Gene Therapy

Evolved riboflavin biosynthesis enzyme RibD as a novel nucleic acid deaminase for base editing.

SUMMARY Enhanced precision in cytosine base editing by employing an evolved riboflavin biosynthesis enzyme RibD that converts targeted cytosines to thymines without intrinsic sequence-context specificity, thereby expanding genomic editing flexibility and enabling more adaptable gene modification tools The Unmet Need: Limitations in current base editors due to sequence-context dependent editing specificity… Read More

A Programmable Translational Activator Platform For Restoring Endogenous Protein Expression In Haploinsufficiency Disorders

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

Nucleic Acid Enrichment And Sequencing Method For Detecting Off-Target Base Editing Events

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

An Engineered Hypercompact CRISPR-Cas12f System with Boosted Gene-Editing Activity

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

Reversible Gene Editing System For Site-Specific Large DNA Integration And Excision

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

Context-Specific Cytosine Base Editors For Precise Single-Nucleotide DNA Editing

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

An Innovative Cutaneous Gene Therapy Platform For Metabolic Disorders

SUMMARY Versatile cutaneous gene therapy platform through skin transplants to stably deliver GLP-1 and other protein factors to treat metabolic disorders   The Unmet Need: Long-lasting, safe, and effective gene therapy treatments for metabolic disorders The field of metabolic disease treatment has long been challenged by the need for precise,… Read More

A Highly-Efficient Platform To Form Vesicular Polymer Nanoparticles For RNA And Protein Payload Delivery

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

In Vitro Assay To Select Optimal Small RNA Guides To Redirect ADAR (Adenosine Deaminase That Acts On RNA) Enzymes

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

Multihydroxy-Propane Vesicle System and Method for Topical Delivery of mRNA

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

Programmed RNA Editing with an Evolved Bacterial Adenosine Deaminase

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

Programmable Translational-Activating RNA Therapeutic Platform Technology

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