TALE-linked recombinant nucleases target DNA breaks while steering repair from NHEJ to HDR for more precise and efficient genome editing.
A Cas9-deaminase-UGI fusion enables precise single-nucleotide editing while avoiding stochastic repair, reducing indels and unwanted mutations.
Mutated Cas9 PAM-interacting residues expand targetable genomic sites while preserving editing precision and lowering off-target activity.
Targeted epigenetic editing silences HBV cccDNA and integrated DNA to suppress replication without DNA cleavage or repair activation.
Targeted epigenetic editing in T cells tunes gene expression to improve ACT expansion, effector function, and persistence.
Targeted epigenetic marks repress HBV regulatory regions in cccDNA and integrated DNA, sustaining viral silencing without DNA breaks.
Inteins self-splice separate TALE and nuclease parts to shrink plasmids while preserving target specificity in genome editing.
Modified ER-LBD fused to caspase-9 enables sustained transgene expression and ligand-triggered apoptosis to improve cell therapy safety.
Donor-template-guided recombinant nucleases shift DNA repair toward HDR, improving genome editing precision without losing cleavage efficiency.
Fusion DNA-binding transactivators let AAV vectors boost SCN1A expression despite limited packaging capacity for gene augmentation.
A nuclease, DNA polymerase, and ssDNA template enable more precise genome sequence replacement with higher editing efficiency and fewer mutations.
Engineered transcription factors and microRNA sites enable selective SCN1A upregulation in parvalbumin neurons while limiting immune response and off-target effects.
Separating CAR recognition and signaling polypeptides enables tunable immune activation, marker switching, and logic-based cancer targeting.
Targeted dCas9 repressor modules silence HBV DNA transcription to cut viral replication and protein levels without genetic disruption.
An engineered DNA polymerase with TBD and thioredoxin cuts STR stutter artifacts without DTT or TCEP, simplifying PCR workflows.
Splitting dCas9 and KRAB across AAV vectors overcomes packaging limits while enabling stable, targeted in vivo gene repression.
A locus-specific CRISPR epigenetic system reactivates maternal 15q11-13 genes while limiting off-target effects through non-viral delivery.
A unique re-editable template sequence enables precise repeat genome edits while limiting unwanted cleavage, off-target effects, and cell-line rework.
Specific APOBEC1 mutations suppress unwanted RNA and DNA edits while preserving Cas9-guided cytidine base editing precision.
Engineered transcription repressors target regulatory DNA and poly(CAG) tracts to durably silence pathogenic alleles with fewer invasive treatments.
Engineered zinc finger, TALE, and CRISPR Htt repressors use AAV9 brain delivery to suppress mutant alleles and reduce HD-related neuronal damage.
Catalytically dead Type V CRISPR fused to repressor domains enables targeted gene silencing with lower off-target effects and flexible gRNA retargeting.
HbW-based editing agents enable precise gene modification without DNA breaks, reducing off-target effects and working in non-replicating cells.