China
Africa
Explore chapters and articles related to this topic
Genome Editing and Gene Therapies: Complex and Expensive Drugs
Published in Peter Grunwald, Pharmaceutical Biocatalysis, 2020
One important application of the CRISPR/Cas system is the CRISPR/dCas9-mediated epigenome editing enabling insight into histone post-translational modifications, non-coding RNAs, and others, responsible for the organization of chromatin structure and epigenetic regulation; it provides information into the role epigenetic regulation has in connection with developing diseases and other biological phenomena (Xie et al., 2018; Enríquez, 2016). For this purpose dCas9 is fused to an epieffector which results in activation (e.g., acetylation of histone lysine residues) or repression (transfer of methyl or removal of acetyl groups from histone residues). An example of an activation has been described by Hilton et al. (2015), who generated an effector fusion protein of dCas9 and the catalytic core of the human acetyltransferase p300 that catalyzed acetylation of histone H3 lysine 27 associated with transcriptional activation of genes from promoters and enhancers (see also Chapter 11).
Lipid-based nanocarrier mediated CRISPR/Cas9 delivery for cancer therapy
Published in Journal of Biomaterials Science, Polymer Edition, 2023
Aisha Aziz, Urushi Rehman, Afsana Sheikh, Mohammed A. S. Abourehab, Prashant Kesharwani
Despite some advancements in recent decades, the number of individuals who die from cancer exemplifies the critical need for novel and also more efficacious alternatives. The quintessential objective of any cancer therapy is to slow tumor progression and growth by correcting mutations and coorect dysregulated expression of genes. The CRISPR/Cas9 gene editing system has been extensively used in basic cancer research, which appears to hold enormous potential in cancer targeted therapy. The regulation of endogenous gene expression is one potential application of the CRISPR/Cas9 system in cancer therapy. Another potential therapeutic application would be to bind dCas9 to histone proteins that participate in DNA methylation to conduct tailored ‘epigenome editing’ [18]. Acute myeloid leukemias are highly malignant neoplasms though they don’t occur frequently. They are responsible for a huge number of deaths due to cancer [19] with five-year survival rate of 24% and unclear etiology. The incidence extends with the age and the median age of diagnosis is 68 years in the US which makes the disease highly poignant [20]. A study was conducted on chronic myeloid leukemia in mice that utilized sgRNA encoding CRISPR/Cas9 plasmid. It targeted the abnormal BCR-ABL gene and thus, improved the survival rate [21].