Therapeutic Genome Editing: Prospects and Challenges
By David B. T. Cox, Randall J. Platt, Feng Zhang. Nature Medicine, 2015
1,224 citations recorded by the source corpus.
Recent advances in the development of genome editing technologies based on programmable nucleases have substantially improved our ability to make precise changes in the genomes of eukaryotic cells. Genome editing is already broadening our ability to elucidate the contribution of genetics to disease by facilitating the creation of more accurate cellular and animal models of pathological processes. A particularly tantalizing application of programmable nucleases is the potential to directly correct genetic mutations in affected tissues and cells to treat diseases that are refractory to traditional therapies. Here we discuss current progress toward developing programmable nuclease–based therapies as well as future prospects and challenges.
Subfields: Molecular Biology
View Paper: https://www.semanticscholar.org/paper/1167a8bd0e718c884c741a88b75286397786eda5
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In Molecular Biology
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Citations in the corpus
- Delivering CRISPR: a review of the challenges and approaches (2018, background): “To date, over 3000 genes have been associated with disease-causing mutations (Cox et al., 2015).”
- Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery. (2019, neutral): “120 With ZFNs, TALENs, and CRISPR-Cas, editing occurs after cellular DNA repair pathways resolve the DNA double-stranded break (DSB) by non-homologous end joining (NHEJ), which can introduce insertions or deletions, or by homology-directed repair (HDR), with a donor sequence present at the site of…”
- Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo (2016, positive): “However, correction of 6% of hepatocytes following a single round of treatment without selection suggests that this approach may be suitable for the treatment of a range of metabolic liver diseases and hemophilia, where restoration of 3–7% normal functional protein can be therapeutic 39 .”