CRISPR-Cas9 has recently emerged as a promising system for multiplexed genome editing as well as epigenome and transcriptome perturbation. Due to its specificity, ease of use and highly modular programmable nature, it has been widely adopted for a variety of applications such as genome editing, transcriptional inhibition and activation, genetic screening, DNA localization imaging, and many more. In this review, we will discuss non-editing applications of CRISPR-Cas9 for transcriptome perturbation, metabolic engineering, and synthetic biology.
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http://dx.doi.org/10.1016/j.copbio.2016.06.003 | DOI Listing |
Proc Natl Acad Sci U S A
January 2025
Innovative Genomics Institute, University of California, Berkeley, CA 94720.
The widespread application of genome editing to treat and cure disease requires the delivery of genome editors into the nucleus of target cells. Enveloped delivery vehicles (EDVs) are engineered virally derived particles capable of packaging and delivering CRISPR-Cas9 ribonucleoproteins (RNPs). However, the presence of lentiviral genome encapsulation and replication proteins in EDVs has obscured the underlying delivery mechanism and precluded particle optimization.
View Article and Find Full Text PDFHum Genet
January 2025
John P. Hussman Institute for Human Genomics, University of Miami Miller School of Medicine, Miami, FL, USA.
Developmental anomalies of the hearing organ, the cochlea, are diagnosed in approximately one-fourth of individuals with congenital. The majority of patients with cochlear malformations remain etiologically undiagnosed due to insufficient knowledge about underlying genes or the inability to make conclusive interpretations of identified genetic variants. We used exome sequencing for the genetic evaluation of hearing loss associated with cochlear malformations in three probands from unrelated families deafness.
View Article and Find Full Text PDFJ Mater Chem B
January 2025
Department of Chemistry, University of North Dakota, Grand Forks, ND, 58202, USA.
Regulation of gene expression in eukaryotic cells is critical for cell survival, proliferation, and cell fate determination. Misregulation of gene expression can have substantial, negative consequences that result in disease or tissue dysfunction that can be targeted for therapeutic intervention. Several strategies to inhibit gene expression at the level of mRNA transcription and translation have been developed, such as anti-sense inhibition and CRISPR-Cas9 gene editing.
View Article and Find Full Text PDFCurr Opin Microbiol
January 2025
Gulbenkian Institute for Molecular Medicine (GIMM), Avenida Professor Egas Moniz, Lisboa, Portugal. Electronic address:
Genome editing technologies, such as CRISPR-Cas9, have revolutionised the study of genes in a variety of organisms, including unicellular parasites. Today, the CRISPR-Cas9 technology is vastly applied in high-throughput screens to investigate interactions between the Apicomplexan parasite Toxoplasma gondii and its hosts. In vitro and in vivo T.
View Article and Find Full Text PDF3 Biotech
January 2025
School of Health Sciences and Technology (SoHST), UPES, Dehradun, Uttarakhand 248007 India.
Triple-negative breast cancer (TNBC) is characterized by a diverse range of molecular features that have been extensively studied. MYC plays a critical role in regulating metabolism, differentiation, proliferation, cell growth, and apoptosis. Dysregulation of MYC is associated with poor prognosis and contributes to the development and progression of breast cancer.
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