The CRlSPR-Cas system is currently the most widely used technology for targeted modifcation of specihc sites in the genome.It employs sgRNA to guide Cas proteins to recognize and cleave thetarget sequence.
Each genome editing tool has its own unique characteristics. Whenapplied to human somatic cells for therapeutic purposes, the editingoutcomes and safety are key concerns for regulatory authorities.
Target |
DNA |
Editing Method |
Double-Strand Break (DSB) |
Features |
NGG PAM recognition; efhcient and versatile |
Advantages |
User-friendly, with a wide range of established applications (e.g., CAR-T, geneticdisease studies) |
Limitations |
Potential for off-target effects; dependent on the cell's DNA repair machinery |
Target |
DNA |
Editing Method |
Double-Strand Break (DSB) |
Features |
NGG PAM recognition; efhcient and versatile |
Advantages |
User-friendly, with a wide range of established applications (e.g., CAR-T, geneticdisease studies) |
Limitations |
Potential for off-target effects; dependent on the cell's DNA repair machinery |
Target |
DNA |
Editing Method |
Double-Strand Break (DSB) |
Features |
NGG PAM recognition; efhcient and versatile |
Advantages |
User-friendly, with a wide range of established applications (e.g., CAR-T, geneticdisease studies) |
Limitations |
Potential for off-target effects; dependent on the cell's DNA repair machinery |
Target |
DNA |
Editing Method |
Double-Strand Break (DSB) |
Features |
NGG PAM recognition; efhcient and versatile |
Advantages |
User-friendly, with a wide range of established applications (e.g., CAR-T, geneticdisease studies) |
Limitations |
Potential for off-target effects; dependent on the cell's DNA repair machinery |
Target |
DNA |
Editing Method |
Double-Strand Break (DSB) |
Features |
NGG PAM recognition; efhcient and versatile |
Advantages |
User-friendly, with a wide range of established applications (e.g., CAR-T, geneticdisease studies) |
Limitations |
Potential for off-target effects; dependent on the cell's DNA repair machinery |
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