Synthetic oligodeoxynucleotides (ODNs) have been used as repair templates in gene-editing applications to insert transgenic sequences into defined genomic loci, albeit with low efficiency. Cells engineered in this way are...
Cytotoxic lymphocytes, including T cells and NK cells, are being developed as allogeneic, “off-the-shelf”, cell therapies for the treatment of hematological and solid tumors. Allogenic lymphocyte therapies face challenges, however,...
Cancer immunotherapy has advanced rapidly over the past two decades, with several autologous chimeric antigen receptor (CAR)-T cell therapies approved for the treatment of hematologic cancers. However, CAR-T cells have...
Gene editing technology, which enables the precision modification of DNA in living cells, is being developed for the treatment of various diseases, including genetic diseases and cancer. Gene editing commonly...
Induced pluripotent stem cells (iPSCs) have emerged as an exciting platform for developing personalized cell therapies. However, clinical applications are limited by the low efficiency of viral and episomal reprogramming...
Sequence-specific gene-editing endonucleases, such as zinc finger nucleases (ZFNs), clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9), and transcription activator-like effector nucleases (TALENs) are being used in the...
Messenger RNA (mRNA) has many applications in biological research and therapeutic development. However, its relatively short half-life limits the use of mRNA in applications where long-term protein translation is desired....
To produce a targeted knock-in, a gene-editing endonuclease is used to create a double strand break (DSB) at a target site in the genome, and a plasmid donor containing a...
Gene editing proteins offer an efficient means of knocking out, inserting and repairing nucleic-acid sequences in living cells. However, while gene-editing proteins can efficiently target pre-determined sequences, they can also...
Autologous engineered cell therapies such as autologous chimeric antigen receptor T-cell (CAR-T) therapies have revolutionized the treatment of hematologic cancers, however they are limited by manufacturing time and variability, the...