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Functional genetic screens are a powerful tool for understanding the genetic underpinnings of biological pathways at a systems level. Through the induction of hundreds to thousands of genome-wide modulations, these screens enable researchers to form hypotheses about genetic associations with normal or disease phenotypes. Causal relationships between the genes and phenotypes are then validated through further screening and experimentation.
High CRISPR knockout efficiencies (80-90% KO frequency) can be quickly and inexpensively generated in a heterogeneous population of cells, referred to as a Knockout Cell Pool. This has enabled more researchers to take advantage of genome engineering in their research without needing to optimize CRISPR protocols.
Gene drives are self-propagating mechanisms by which desired genetic variants can be spread through a population faster than traditional Mendelian inheritance. CRISPR is making this strategy so effective that alleles can continue their spread if they confer disadvantageous traits, such as sterility, to an organism.
One of the most widely-used applications of iPS cells is disease modeling. Using the powerful gene-editing tool, CRISPR, researchers can introduce specific mutations to healthy cells to recapitulate diseases. Alternatively, one can revert the genotype of cells taken from individuals with a disease to wild type.
Several researchers use plasmid transfection for their CRISPR genome editing experiments, but is that the best strategy? High off-target effects, variable editing efficiencies, and integration in host genome are just a few of the reasons why RNPs have quickly become the more efficient alternative to plasmids.
CRISPR is a powerful molecular tool that can be used to make a number of different genomic modifications. One of the most common uses of CRISPR is to knock out a gene of interest so that no functional protein is produced. This technique is often used to elucidate the role that genes and their products play in biological processes and disease pathways.
A significant amount of biomedical research relies on the use of immortalized cell lines, which are easy to use and manipulate. However, these cell lines are often genetically abnormal and may not faithfully recapitulate the characteristics of the tissue they are intended to represent. Takahashi and Yamanaka’s 2006 work on induced pluripotent stem (iPS) cells was a seminal innovation in the biomedical field.
CRISPR is an immensely powerful research tool that has revolutionized the way scientists manipulate genomes. In particular, CRISPR-mediated gene knockouts play key roles in drug discovery, understanding gene function, and other uses that improve the world around us.
The drug discovery process, in which compounds are screened and evaluated for therapeutic use, has resulted in safe and effective therapies for a variety of diseases. However, assessments of new compounds for drug development are notoriously long and costly; they typically span more than a decade and exceed a billion dollars.
Genome editing involves the deletion, insertion, or modification of specific DNA sequences in the genome. For many years, researchers had been trying to develop easy and cost-effective genome editing tools to address problems across a wide spectrum of fields. For instance, gene therapy in humans could progress rapidly if one could simply eliminate the gene responsible for a certain genetic disorder.