In Vivo Expansion of Gene-Targeted Hepatocytes through Transient Inhibition of an Essential Gene

Medicine, Hepatobiliary System, Life Sciences, Bioengineering, Genetics,
gene therapy   hepatocytes   in vivo expansion   essential gene   genome editing  

Breakthrough in Gene Therapy: Repair Drive Technology Enables In Vivo Expansion of Hepatocytes Academic Background Gene therapy has become a hot topic in medical research in recent years, especially for liver diseases. Due to the central role of the liver in metabolism, it has become a critical target for research. Although existing gene-editing te...

Superior Fidelity and Distinct Editing Outcomes of SaCas9 Compared with SpCas9 in Genome Editing

Medicine, Hematology, Life Sciences, Cell Biology, Biochemistry, Bioengineering, Genetics,
genome editing   sacas9   spcas9   PAM sequence   insertion efficiency   off-target effects  

Comparison of SaCas9 and SpCas9 in High-Fidelity and Different Gene Editing Outcomes Research Background CRISPR systems based on Cas9 protein have become powerful tools for genome editing, widely used in basic research and clinical gene therapy. Currently, the most commonly used Cas9 variants are SpCas9 from Streptococcus pyogenes and SaCas9 from S...

Development of Multiplexed Orthogonal Base Editor (MOBE) Systems

Life Sciences, Bioengineering, Genetics,
multiplex editing   orthogonal base editors   genome editing   point mutations   high-fidelity Cas9  

In recent years, with the rapid development of gene editing tools, especially the introduction of the CRISPR-Cas9 system, it has become possible to precisely modify specific DNA sequences. Currently, the introduction of single-nucleotide variants (SNVs) as a powerful tool for studying gene function and disease associations is particularly important...

Tracking-seq Reveals the Heterogeneity of Off-target Effects in CRISPR-Cas9-mediated Genome Editing

Medicine, Basic Medical Sciences, Life Sciences, Immunology, Genetics, Biophysics, Microbiology,
CRISPR-Cas9   off-target effects   genome editing   Tracking-seq   genome editing tools  

Transcriptomics Technology Tracking-Seq Reveals Off-Target Effect Heterogeneity Mediated by CRISPR-Cas9 Research Background With the rapid development of genome editing technologies, the CRISPR-Cas9 system has been widely used in biomedical research due to its high efficiency and ease of operation. However, a significant issue with the CRISPR-Cas9 ...