Innovative cell lines enabling precise genetic modifications to advance research in gene function, disease modeling, and potential therapeutic interventions.

A revolutionary drug modality for the selective modification and degradation of intracellular proteins in lysosomes.

Researchers at the University of California, Davis has developed a microRNA-based treatment for traumatic brain injury.

A novel method for selectively targeting and modulating brain border-associated myeloid cells for the treatment of neurological disorders.

Precise control of wound healing depends on physician’s evaluation, experience. Physicians provide conditions and time for body to either heal itself, or to accept and heal around direct transplantations, and their practice relies a lot on passive recovery. Slow healing of recalcitrant wounds is a known persistent problem, with incomplete healing, scarring, and abnormal tissue regeneration. 23% of military blast and burn wounds do not close, affecting a patient’s bone, skin, nerves. 64% of military trauma have abnormal bone growth into soft tissue. While newer static approaches have demonstrated enhanced growth of non-regenerative tissue, they do not adapt to the changing state of wound, thus resulting in limited efficacy.

A cutting-edge vaccine delivery platform that enhances tumor treatment by co-delivering MHC class I and II restricted antigens.

The targeted intracellular delivery of protein cargos is critical for therapeutic applications such as enzyme inhibition, transcriptional modulation, and genome editing. For most tissues, the delivery of these molecules must occur in-vivo. This has historically been achieved using viral vectors or lipid nanoparticles. While significant progress has been made in engineering the tropisms of these particles towards different tissues, delivery specificity and packaging limits remain challenging. UC Berkeley researchers have developed engineered immune cells that produce and intercellularly transfer a protein and/or RNA cargo in response to contact with a predetermined antigen. Proof of concept experiments demonstrated that production of EDVs can be induced in a T cell line through either the presence of a small molecule or recognition by the T cells of a specific antigen on co-cultured cells. The researchers showed that delivery can be achieved using multiple strategies and that the system is compatible with multiple cargo proteins of interest, including Cre recombinase and S.pyogenes Cas9. 

A groundbreaking method for the efficient isolation and preservation of high-purity small extracellular vesicles (sEVs - exosomes) from biofluids using a novel EXO-PEG-TR reagent.

This technology introduces a novel class of synthetic genetic polymers, capable of enhancing protein target binding and mimicking antibodies, for therapeutic and diagnostic applications.

Researchers at UCI and Harvard have engineered a new platform for diversifying antibody genes in yeast, eliminating a crucial bottleneck in making effective antibodies. This technology enables the rapid continuous directed evolution of affinity reagents for applications ranging from structural and cellular biology to diagnostics and immunotherapy.

An innovative small molecules therapy that significantly lowers intraocular pressure in glaucoma patients, offering neuroprotection and addressing trabecular meshwork fibrosis.

This technology offers a significant improvement in the therapeutic application of type E botulinum neurotoxin (BoNT/E) by introducing rationally designed mutations into the receptor binding domain.

IS110 family transposons encode a protein component (also referred to as the transposase) and a non coding RNA component (also referred to as the bridgeRNA or bRNA). In its naturally occurring context, a bRNA-bound transposase directs the integration of its cognate transposon (also referred to as the donor) into target DNA sites. The nucleic acid sequence and structure of the bRNA partially determines the sequence identify of the terminal ends of the mobilized donor, and the sequence identify of the target DNA molecule (also referred to as the target or target DNA). UC Berkeley researchers have developed a programmable gene editing technology based on IS110 family transposons that can be used for targeted insertions, deletions, excisions, inversions, replacements, and capture of DNA in vitro and in vivo. Additionally, this technology can be multiplexed to achieve complex assembles of multiple fragments of DNA.

Efficient delivery and expression of exogenous proteins in cell populations (e.g., cells in the body) for gene therapy / gene editing applications, is an important goal in biomedicine. This can be hampered by inefficient transport of enzymes from outside the body to cells within the body. When delivering nucleic acids or proteins of interest (e.g., DNA editing enzymes), most delivery methods can only reach and enter a small subset of cells within a tissue. There is a need for compositions and methods for improved delivery of proteins of interest, and such is provided herein. UC Berkeley researchers have discovered that delivery of a molecular cargo to a target cell can be more efficiently achieved by using a cell as the delivery vehicle. This can be accomplished by delivering a nucleic acid encoding an enveloped delivery vehicle (EDV) (one that comprises a molecular cargo), to a producer cell where the producer cell produces the EDV and thereby delivers the molecular cargo to neighboring cells (referred to herein as receiver cells). Thus, there is no human intervention between delivery of a subject nucleic acid (encoding the EDV) and subsequent delivery of EDVs to target cells (receiver cells).  

A novel antimicrobial approach combining pore-forming agents with histones to eradicate bacteria and bypass known resistance mechanisms.

SUMO inhibitors offer a promising new therapy for protecting against cardiac rhythm disturbances and pump failure associated with heart attacks.

Researchers at the University of California, Davis have developed a technology that targets CD147 to significantly improve bone health and treat musculoskeletal diseases without the side effects of current therapies.

Existing RNA-targeting tools for sequence-specific manipulation include anti-sense oligos (ASOs), designer PUF proteins and CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas systems. However, there are significant limitations to each of the current tools. ASOs are usually not available for most RNA manipulations other than gene silencing. Designer proteins, such as PUF (Pumilio and FBF homology protein), possess low RNA recognition efficiency and it remains challenging to target RNA sequences >8-nucleotides (nt) in length. The bulky Cas protein (Cas13d: average 930 amino acids) leads to complication for transgene delivery and concerns of its immunogenicity due to its bacterial origin. Mutants of zinc finger(ZnF) proteins in ZRANB2 recognize a single-strand RNA containing a novel GGG motif with micromolar affinity, compared to the original motif GGU. These mutants serve as a foundation for RNA-binding ZnF designer protein engineering for in vivo RNA sequence-specific targeting.ZnFs are generally compact domains (~3kDa each) that have been successfully engineered for DNA recognition as modular arrays. A ZnF-based system has unique advantages, especially in a therapeutic context: (1) Broad application with the possibility to fuse with other effector domains; (2) High efficiency of RNA recognition (3 RNA bases recognized per 30-amino-acid ZnF) with a small size of protein. Only 4 ZnFs (~100 aa) is required for specific targeting in the transcriptome. (3) Humanized components without immunogenic concern.By engineering new sequence specificity of the ZRANB2 ZnF1, researchers from UC San Diego identified 13 mutants that altered their preferred RNA binding motif from GGU to GGG. They are N24R, N24H, N14D/N24R, N14D/N24H, N14R/N24R, N14R/N24H, N14H/N24R, N14H/N24H, N14Q/N24R, N14Q/N24H, N14E/N24R, N14S/N24R, N14E/N24H.

Lipid Nanoparticles (LNPs) are a leading platform for nucleic acid delivery, widely used in therapeutics and vaccine development. However, the process of optimizing new LNP formulations has been significantly hindered by labor-intensive and costly screening methods, which require individual injections into animal models. Given the vast array of potential lipid compositions and formulation variables, these constraints severely impede the efficiency of research and development.To overcome these challenges, UC Berkeley researchers have developed a novel approach for identifying and characterizing functional nucleic acid delivery vehicles. This innovative method leverages circular RNA barcoding technology, enabling a more efficient screening process. Instead of relying on conventional cell sorting techniques, which restrict screening to specific organs and host species, this breakthrough allows direct detection of barcoded nucleic acids within circular RNAs in treated cells. By analyzing the barcodes detected, researchers can accurately determine which lipid compositions and formulations successfully delivered RNA molecules.  This technology represents a significant advancement in LNP research, offering a scalable, cost-effective solution that enhances the precision and scope of nucleic acid delivery screening.

Researchers at UC Irvine have identified genetic polymorphisms associated with disease progression and responsiveness to treatment with Tetracosapentaenoic acid (24:5 n-3) for age-related eye disorders such as age-related macular degeneration (AMD), diabetic retinopathy and glaucoma. These variations found in the ELOVL2 gene are associated with AMD progression and the varying responses individuals have to AMD treatments, including preventative measures. Additionally, these genetic variations have applications in human identification.

Researchers at the University of California, Davis, have developed a process for isolating anti-inflammatory lipids for treating autoimmune and inflammatory diseases.

Researchers at the University of California, Davis have developed a biologic dressing derived from fish skin to enhance wound healing.

Influenza A virus (IAV) poses an ever-evolving threat due to its high mutation rate and ability to reassort, leading to new viral variants that evade existing vaccines and treatments. Historically responsible for devastating global pandemics, including the infamous Spanish Flu, and currently fueling concerns with the spread of highly pathogenic Avian Influenza (HPAI H5N1), IAV remains a pressing global health challenge.UC Berkeley researchers have developed an Antisense Oligonucleotides (ASO) therapy that is an next-gen approach to combating influenza by modulating IAV activity at its genetic level. Unlike traditional antivirals or seasonal vaccines that struggle to keep up with mutating strains, this ASOs therapy targets the ultra-conserved U12 region within the IAV RNA genome, offering broad-spectrum efficacy against even the most elusive influenza strains.  

Precise control of wound healing depends on physician’s evaluation, experience. Physicians provide conditions and time for body to either heal itself, or to accept and heal around direct transplantations, and their practice relies a lot on passive recovery. Slow healing of recalcitrant wounds is a known persistent problem, with incomplete healing, scarring, and abnormal tissue regeneration. 23% of military blast and burn wounds do not close, affecting a patient’s bone, skin, nerves. 64% of military trauma have abnormal bone growth into soft tissue. While newer static approaches have demonstrated enhanced growth of non-regenerative tissue, they do not adapt to the changing state of wound, thus resulting in limited efficacy.

Precise control of wound healing depends on physician’s evaluation, experience. Physicians provide conditions and time for body to either heal itself, or to accept and heal around direct transplantations, and their practice relies a lot on passive recovery. Slow healing of recalcitrant wounds is a known persistent problem, with incomplete healing, scarring, and abnormal tissue regeneration. 23% of military blast and burn wounds do not close, affecting a patient’s bone, skin, nerves. 64% of military trauma have abnormal bone growth into soft tissue. While newer static approaches have demonstrated enhanced growth of non-regenerative tissue, they do not adapt to the changing state of wound, thus resulting in limited efficacy.