A highly precise and efficient gene-editing tool designed to correct single-nucleotide DNA mutations responsible for genetic diseases.

Brief description not available

Brief description not available

Brief description not available

Brief description not available

Brief description not available

Brief description not available

Researchers at the University of California, Davis have developed a technology that introduces an approach to creating semi-living, non-replicating cellular systems for advanced therapeutic applications.

Progressive Multifocal Leukoencephalopathy (PML) is a devastating and often fatal demyelinating disease of the central nervous system caused by the reactivation of the JC virus (JCV). In immunocompromised patients, the absence of effective T cell surveillance allows the virus to infect and lyse oligodendrocytes, leading to irreversible neurological damage. UC Berkeley researchers have developed a method for discovering and engineering antigen-specific T cell receptors (TCRs) that specifically target JCV.

Researchers at the University of California, Davis have developed novel antisense oligonucleotide (ASO) therapies that enhance ADNP protein expression to address haploinsufficiency in ADNP syndrome.

Researchers at the University of California, Davis have engineered dominant negative CD40L mutant polypeptides that inhibit CD40/CD40L-mediated signaling, offering therapeutic potential for inflammatory, immune disorders, and cancer with improved safety profiles.

Clustered regularly interspaced short palindromic repeats (CRISPR) screening is a cornerstone of functional genomics, enabling genome-wide knockout studies to identify genes involved in specific cellular processes or disease pathways. The success of CRISPR screens depends critically on the design of effective guide RNA (gRNA) libraries that maximize on-target activity while minimizing off-target effects. Current CRISPR screening lacks tools that can natively integrate next-generation sequencing (NGS) data for context-specific gRNA design, despite the wealth of genomic and transcriptomic information available from modern sequencing approaches. Traditional gRNA design tools have relied on static libraries with limited genome annotations and outdated scoring methods, lacking the flexibility to incorporate context-specific genomic information. Off-target effects are also a concern, with CRISPR-Cas9 systems tolerating up to three mismatches between single guide RNA (sgRNA) and genomic DNA, potentially leading to unintended mutations that could disrupt essential genes and compromise genomic integrity. Additionally, standard CRISPR library preparation methods can introduce bias through PCR amplification and cloning steps, resulting in non-uniform gRNA representation.

This technology represents a groundbreaking approach to treating Primary Open Angle Glaucoma by directly targeting the trabecular meshwork pathology with lipid nanoparticle-mediated delivery of gene editing tools or anti-sense oligos.

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

Achieving precise and tunable control over endogenous gene expression in eukaryotic cells remains a significant challenge, particularly for therapeutic applications or detailed biological studies where fine-tuning is required rather than complete on/off switching. This innovation, developed by UC Berkeley researchers, addresses this by providing a novel, programmable method for transcriptional tuning. The innovation is a two-domain fusion protein comprising the transcriptional repression domain (TRD) of the methyl-CpG-binding domain (MBD) protein MeCP2 linked to a dead Cas9 (dCas9) domain. When combined with a single guide RNA (sgRNA) that targets a specific endogenous gene, this fusion protein partially inhibits, or "tunes," the expression of that gene. Unlike traditional methods like RNAi or full CRISPR interference (CRISPRi), which often aim for complete knockdown, this system offers a highly specific and titratable way to dial down gene expression, providing a distinct advantage in studies requiring subtle modulation of gene dosage or for developing dose-dependent therapeutic strategies.

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

This technology represents a pioneering approach to vaccine development, focusing on encapsulated adjuvants and antigens to enhance efficacy while minimizing side effects.

Tissue targeting and cargo packaging limitations are two of the most challenging barriers to in-vivo therapeutic delivery. Overcoming both of these issues, UC Berkeley researchers have developed engineered immune cells that produce enveloped delivery vehicles (EDVs) capable of encapsulating protein and/or RNA therapeutics that can be delivered to a target cell with a predetermined trigger. Triggers can either be the presence of a small molecule, or recognition of a specific antigen on the target cell. The researchers showed that delivery can be achieved in a co-cultured system using various strategies and that the system is compatible with multiple cargo proteins of interest including Cre recombinase and RNA-complexed Cas proteins. This technology opens possibilities for broader and safer in-vivo therapeutic delivery.

A revolutionary one-shot therapy for juvenile and adult-onset glaucoma affected by MYOC mutations, offering a permanent cure for this previously untreatable disease.

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.

Most tumors are extremely complex, having many oncogene drivers and are, therefore, not as amenable to a CRISPR-mediated therapies. Pediatric low-grade glioma (pLGG) is a type of brain cancer that arises during childhood. Some interventions exist, including surgery and inhibitor drugs, but there is no cure for pLGG. In contrast to most types of cancer (which feature a host of driver oncogenes), pLGG tumors tend to arise due to a single driver oncogene mutation. This aspect makes pLGG a potential target for a genome editing intervention. Because CRISPR enzymes can precisely discriminate between wild-type and mutant sequences in a single cell, enzymes such as Cas9 can target a mutant oncogene site without impacting the corresponding wild-type locus in a non-cancer cell. UC Berkeley researchers have developed a CRISPR-based strategies for anti-cancer genome editing.  The invention consists of a suite of genome editing strategies with the capacity to selectively inactivate the oncogene underlying tumor pathology, for example, mutations in pLGG. Deployed via a delivery strategy with the capacity for broad genome editing of brain cells, our strategy will have the capacity to halt – and potentially reverse – tumor growth.

A novel dendronized polypeptide architecture for efficient and safe mRNA delivery, suitable for anti-tumor immunotherapy.

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

A novel biological therapy that targets the ataxin-3 mRNA to treat ALS by increasing ataxin-3 protein levels.

Professor Giulia Palermo and colleagues from the University of California, Riverside and the University of Rochester have developed high-fidelity Cas13a variants with increased sensitivity for base pair mismatches.The activation of these Cas13a variants can be inhibited with a single mismatch between guide-RNA and target-RNA, a property that can be used for the detection of SNPs associated with diseases or specific genotypic sequences.