CRISPR-based genome editing in disease treatment

CRISPR-based genome editing tools used for genetic disease treatment mainly include Cas nucleases, base editors (BEs), and prime editors (PEs). High-throughput functional screenings enable accurate dissection of complex proteins, and identification of single nucleotide variants (SNVs) within the same gene, including those that confer sensitivity or resistance to drugs. Efficient delivery of genome editing machinery to target cells or tissues is crucial for successful genome modification either in or outside the body. Viral vectors such as lentivirus (LV), adenovirus (Adv), and adeno-associated virus (AAV) offer complementary advantages, and non-viral delivery provides the advantage of transient editing activity.