Engineered Viruses as Genome Editing Devices

Engineered Viruses as Genome Editing Devices

Genome editing based on sequence-specific designer nucleases, also known as programmable nucleases, seeks to modify in a targeted and precise manner the genetic information content of living cells. Delivering into cells designer nucleases alone or together with donor DNA templates, which serve as surrogate homologous recombination (HR) substrates, can result in gene knockouts or gene knock-ins,...

Advancing Chimeric Antigen Receptor-Engineered T-Cell Immunotherapy Using Genome Editing Technologies: Challenges and Future Prospects

Chimeric antigen receptor engineered-T (CAR-T) cells also named as living drugs, have been recently known as a breakthrough technology and were applied as an adoptive immunotherapy against different types of cancer. They also attracted widespread interest because of the success of B-cell malignancy therapy achieved by anti-CD19 CAR-T cells. Current genetic toolbox enabled the synthesis of CARs ...

Genome editing with engineered nucleases in plants.

Numerous examples of successful 'genome editing' now exist. Genome editing uses engineered nucleases as powerful tools to target specific DNA sequences to edit genes precisely in the genomes of both model and crop plants, as well as a variety of other organisms. The DNA-binding domains of zinc finger (ZF) proteins were the first to be used as genome editing tools, in the form of designed ZF nuc...

Genome Editing



Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing

Genome editing with CRISPR/Cas9 is a promising new approach for correcting or mitigating disease-causing mutations. Duchenne muscular dystrophy (DMD) is associated with lethal degeneration of cardiac and skeletal muscle caused by more than 3000 different mutations in the X-linked dystrophin gene (DMD). Most of these mutations are clustered in "hotspots." There is a fortuitous correspondence bet...