Using genome editing to engineer universal platelets

Precise and heritable genome editing in evolutionarily diverse nematodes using TALENs and CRISPR/Cas9 to engineer insertions and deletions.

Exploitation of custom-designed nucleases to induce DNA double-strand breaks (DSBs) at genomic locations of choice has transformed our ability to edit genomes, regardless of their complexity. DSBs can trigger either error-prone repair pathways that induce random mutations at the break sites or precise homology-directed repair pathways that generate specific insertions or deletions guided by exo...

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 using NucleofectorTM Technology

Genome editing using Cas9 nickases.

The RNA-guided, sequence-specific endonuclease Cas9 has been widely adopted as genome engineering tool due to its efficiency and ease of use. Derived from the microbial CRISPR (clustered regularly interspaced short palindromic repeat) type II adaptive immune system, Cas9 has now been successfully engineered for genome editing applications in a variety of animal and plant species. To reduce pote...

Genome Editing Using Mammalian Haploid Cells

Haploid cells are useful for studying gene functions because disruption of a single allele can cause loss-of-function phenotypes. Recent success in generating haploid embryonic stem cells (ESCs) in mice, rats, and monkeys provides a new platform for simple genetic manipulation of the mammalian genome. Use of haploid ESCs enhances the genome-editing potential of the CRISPR/Cas system. For exampl...