CRISPR, crispr genome editing system - Part 14

Controlling CRISPR

As our understanding of CRISPR technology grows, the extraordinary potential of this gene-editing system in the realm of therapeutic development comes closer and closer to realization. However, ownership of the intellectual property (IP) behind this technology is set to spark a protracted patent feud between the most prominent scientists in the field. Who will reap the financial rewards if CRISPR revolutionizes the pharmaceutical industry and launches a new era of human medicine? CRISPR holds incredible therapeutic promise The CRISPR/Cas9 system holds several advantages over existing gene-editing technologies. Firstly, RNA-guided gene targeting via CRISPR/Cas9 is fundamentally easier and vastly cheaper when compared with Zinc-finger nucleases or TALENs, both of which require recombinant protein engineering. Experimentally, the system has been simple enough...
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CRISPR Chain Reaction

A rare mosaic female fly, with a lighter left half mutated by MCR and a wild-type darker right half. A new genetic-editing technique based on integrating CRISPR/Cas9 technology into a Drosophila melanogaster genome can make homozygous mutants in half the time it would take using traditional crosses, according to a paper published today (March 19) in Science. “The study is well done and also very elegant,” said Ji-Long Liu of the University of Oxford who was not involved in the research, but helped to develop CRISPR/Cas9 in Drosophila. Liu called the method “a really clever way to . . . make the magic happen.” The CRISPR/Cas9 system requires two components: a guide RNA that matches the region of the genome...
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A CRISPR Solution to ‘Bubble Boy’ Disease?

They named him Phoenix because he was born five weeks early while his parents were on vacation, and spent his first few weeks in an incubator. Kristen and Patrick Wilkinson thought they knew exactly which ashes their son might soon rise from. But when they got him home to San Francisco things just got worse, Kristen says. Phoenix wasn’t gaining weight. He had a persistent skin rash. Eventually he was admitted to UCSF Benioff Children’s Hospital with a diagnosis of “failure to thrive.” Phoenix had been born in Kentucky, a state where, unlike in California, infants are not routinely screened for a disease called SCID — Severe Combined Immunodeficiency. So at first, California doctors puzzled over what might be wrong...
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Microbiome Editing with CRISPR/Cas9 Has Research, Diagnostic Potential

NEW YORK (GenomeWeb) – Two of the bacterial kingdom’s greatest assets, the CRISPR “immune” system and horizontal gene transfer of plasmids by conjugation, could be exploited by humans to wipe out specific communities of bacteria or even turn them into useful reporters. CRISPR/Cas9 provides a way to kill off bacterial populations according to the genetic sequences in their genomes, according to a paper published in Nature Biotechnology in September. The authors showed that they could selectively eliminate populations of bacteria harboring antibiotic resistance genes by targeting them with Cas9 nucleases informed by guide RNAs that corresponded to those genes. They did so by creating plasmids packing the Cas9 enzyme and relevant guide RNAs. “We can selectively cleave DNA in bacteria...
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Building a genomic GPS with CRISPR

A new “app” for finding and mapping chromosomal loci using multicolored versions of CRISPR/Cas9, one of the hottest tools in biomedical research today, has been developed by scientists at UMass Medical School. This labeling system could be a key to understanding the spatial and temporal regulation of gene expression by allowing researchers to measure the precise linear distance between two known points on different chromosomes or two locations on the same chromosome in live human cells. Detail of the findings were published in Proceedings of the National Academy of Sciences (PNAS) and first presented at the American Society for Cell Biology–International Federation for Cell Biology annual meeting in December. The nucleus of every cell in our bodies (with the exception...
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Scientists Call for a Summit on Gene-Edited Babies

A group of senior American scientists and ethics experts is calling for debate on the gene-engineering of humans, warning that technology able to change the DNA of future generations is now “imminent.” In policy recommendations published today in the journal Science, eighteen researchers, including two Nobel Prize winners, say scientists should accept a self-imposed moratorium on any attempt to create genetically altered children until the safety and medical reasons for such a step can be better understood. The concern is over a rapidly advancing gene-editing technology, called CRISPR-Cas9, which is giving scientists the ability to easily alter the genome of living cells and animals (see “Genome Surgery”). The same technology could let scientists correct DNA letters in a human embryo...
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Biologists devise invasion plan for mutations

Biologists devise invasion plan for mutations On 28 December 2014, Valentino Gantz and Ethan Bier checked on the fruit flies that had just hatched in their lab at the University of California (UC), San Diego. By the classic rules of Mendelian genetics, only one out of four of the newborn flies should have shown the effects of the mutation their mothers carried, an X-linked recessive trait that causes a loss of pigmentation similar to albinism. Instead, nothing but pale yellow flies kept emerging. “We were stunned,” says Bier, who is Gantz’s Ph.D. adviser. “It was like the sun rose in the west rather than the east.” They hammered out a paper and submitted it to Science 3 days later. In...
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