Gene Editing Enzymes & Reagents
Explore our GMP-Grade CRISPR Cas9, Cas12a, MaxNuclease, AAV ELISA Kits, and DNA Amplification Enzymes
Gene therapy generally functions by introducing exogenous genes (or gene-editing tools) into target cells or tissues, serving to replace, compensate, block, correct, augment, or knockout specific genes to exert therapeutic effects . Based on the different methods of introducing genes into the human body, gene therapy can be divided into ex vivo therapies (extracorporeal therapies) and in vivo therapies (intracorporeal therapies). Ex vivo therapy generally involves introducing exogenous genes (or gene-editing tools) into cells outside the body, preparing them to become genetically modified cells or cell-derived products, and ultimately reintroducing them to the body to exert therapeutic effects. In vivo therapy introduces exogenous genes (or gene-editing tools) into the human body via non-viral vectors (such as LNP) or viral vectors (such as Adeno-Associated Virus AAV, Lentivirus LV, etc.) to exert therapeutic effects.
Figure 1. Types of gene editing .
GMP Cas9 & Cas12a Nucleases
Cas9 and Cas12a are primarily used for the genetic modification of cells and gene therapy drugs, including hematopoietic stem cells and T cells. Our GMP-Grade Cas9 nuclease is manufactured in a high-standard GMP-Grade enzyme production facility and has undergone rigorous in vitro and intracellular quality validation. KACTUS has filed our GMP Cas9 with the FDA Drug Master Files (DMF# 036578). KACTUS has provided regulatory support to customers completing Investigational New Drug (IND) applications with our Cas9.
Figure 2. (Left) Cas9 Nuclease is used for gene knockout in 293T cells, Jurkat cells, and T cells, and its gene knockout efficiency in various cell types is comparable to that of leading suppliers. (Right) Cas9 Nuclease In Vitro DNA Cleavage Experiment. The in vitro cleavage efficiency of Cas9 nuclease is equivalent across all three batches.
Cas9 ELISA Kit
KACTUS Cas9 ELISA kit is for quantitative detection of residual Cas9 nuclease in gene-edited cell drugs before reinfusion into the human body, whether extracellular or intracellular. Our test kit has a detection range of 0.25 ng/mL to 16 ng/mL, with sensitivity reaching up to 0.125 ng/mL.
High Accuracy and Standard Curve R² > 0.99
Figure 3. (Left) Cas9 ELISA Kit example standard curve with an R² of 0.9997. (Right) The same test kit was used to assess the spike-recovery rate of Cas9 nuclease at three different concentrations (high, medium, low) in dilution buffer. The spike-recovery rate for different concentrations of Cas9 samples in dilution buffer all fell between 80%-120%, indicating that this ELISA kit has high accuracy in detecting residual Cas9 nuclease.
 Technical Guidance Principles for the Pharmacological Research and Evaluation of In Vivo Gene Therapy Products (Trial)
 Savić N, Schwank G. Advances in therapeutic CRISPR/Cas9 genome editing. Transl Res.