CRISPR, crispr genome editing system - Part 4

Crispr goes commercial: Genetic goldrush

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Within just three years since the discovery of its gene-editing potential, the new technique Crispr has become the hottest, and most controversial, development in genomics research. And now it’s more than just a science – it’s big business too. First discovered in bacteria, Crispr (clustered regularly interspaced short palindromic repeats) is a genome-editing tool that can target specific genes in any organism based on RNA–DNA base pairing and then precisely cut the gene through the activities of the enzyme known as Crispr-associated protein 9 or Cas9. The technology can delete, repair or replace genes, and is faster, easier, cheaper and – in principle – more precise than other gene-editing techniques. With potential benefits across human health, agriculture and industrial biotechnology,...
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Molecular biology meets computer science tools in new system for CRISPR

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Nicolo Fusi is one of the researchers who collaborated on the new CRISPR tools A team of researchers from Microsoft and the Broad Institute of MIT and Harvard has developed a new system that allows researchers to more quickly and effectively use the powerful gene editing tool CRISPR. The system, unveiled Monday and dubbed Azimuth, uses machine learning, in which a computer takes a limited set of training data and uses that to learn how to make predictions about data it hasn’t yet seen. In this case, the machine learning system is being used to predict which part of a gene to target when a scientist wants to knockout – or shut off – a gene. Machine learning enables the...
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Controversial CRISPR history sets off an online firestorm

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It isn’t often that biology finds itself in the same galaxy of the Twitterverse as, say, “The Bachelor,” but on Tuesday morning CRISPR did. The trending topic was sparked by a recent “Perspective” in Cell limning the history of the gene-editing technique. But this history was loaded, as history often is. CRISPR’s history could determine the outcome of a bitter patent fight worth many millions of dollars, and this version was written by the director of the institute that stands to reap some of those millions. The essay has triggered a deep controversy over disclosing conflicts of interest and the close ties between journals and certain research groups. CRISPR is a tool that acts as a microscopic pair of scissors with the ability to slice DNA. The Perspective’s author, Eric Lander, is president...
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Doctors Might One Day Treat Cancer By Cutting Bad DNA

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Gene editing grabbed headlines recently after the discovery of CRISPR, a tool that allows scientists to cut out a particular, potentially faulty gene and paste another one in its place. Dozens of other gene-editing technologies existed before CRISPR. French biotechnology company Cellectis was experimenting with one such technique, called TALENs. Using the tool, Cellectis is developing treatments for cancers, and last year as part of a rare exception to a limited clinical trial, they treated an 11-month-old girl named Layla who had otherwise untreatable leukemia. Business Insider sat down with Cellectis CEO Andre Choulika to learn more about TALENs and his plans toward getting more treatments like the one that helped Layla approved. TALENs, the trademarked acronym that stands for...
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Intellia Therapeutics Launches New Division to Accelerate Ex Vivo Programs with CRISPR/Cas9

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Intellia Therapeutics has launched a new division, eXtellia Therapeutics, with the intent of focusing resources and research on ex vivo applications of the novel technology, CRISPR/Cas9. As in vivo and ex vivo programs require different competencies in research, manufacturing and commercialization, eXtellia Therapeutics is being launched to accelerate Intellia’s efforts in areas of significant unmet medical need – immuno-oncology, autoimmune and inflammatory diseases – using an ex vivo approach. Intellia will continue its in vivo programs and strategy through a dedicated scientific team. “We are excited to announce the establishment of eXtellia, increasing our footprint in ex vivo gene editing for oncology and autoimmune diseases,” said Nessan Bermingham, Ph.D., Chief Executive Officer and Founder, Intellia Therapeutics. “eXtellia enables us to...
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U.K. researcher details proposal for CRISPR editing of human embryos

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LONDON—The hottest technique in gene editing, called CRISPR, could soon be used to study human embryos. On 14 January, a regulatory committee in the United Kingdom will evaluate a request to knock out development genes in day-old embryos. In a press conference today, Kathy Niakan, a researcher at the Francis Crick Institute in London, discussed the rationale behind her proposed project and the hope that this line of inquiry might one day improve treatments for infertility. Niakan studies how the single cell of a fertilized egg turns into a blastocyst, the approximately 5-day-old structure that subsequently implants in the mother’s uterus. The blastocyst contains several types of cells. Those destined to become the fetus are called epiblast progenitor cells. They...
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The CRISPR tool is already so crucial … that it was nominated for Word Of The Year – Washington Post

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A futuristic gene-editing technology holds almost fantastical promise to turn biologists’ wildest dreams into reality. The technology called CRISPR could one day be used to delete the HIV virus from patients’ cells, create a limitless supply of organs for transplant and produce better crops. Already, it’s being used in laboratories across the world. This week, the fierce battle over who invented it officially kicked off, with hundreds of millions of dollars on the line. A patent judge rang the opening bell Monday, pitching two heavyweight research institutions perched on opposite coasts against each other: the University of California Berkeley vs. the MIT- and Harvard-affiliated Broad Institute in Cambridge. The judge’s declaration of “interference,” a technical term meaning that a conflict exists between a patent application and another...
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‘Crispr’ gene editing technique may treat inherited blindness

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New York: A new gene editing technique which was hailed as the breakthrough of 2015 by Science Magazine has been found effective in preventing retinal degeneration in a type of inherited blindness, a study says. The researchers focused on inherited retinitis pigmentosa, a degenerative eye disease with no known cure that can lead to blindness. They used a technique known as Crispr-Cas9, to remove a genetic mutation that causes the blindness disease. Although the study involved rats, it is an important milestone because of its potential implications for humans. “Our data show that with further development, it may be possible to use this gene-editing technique to treat inherited retinitis pigmentosa in patients,” said senior author of the study Shaomei Wang...
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Finding Enhancers with CRISPR

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While several groups are working to apply the CRISPR/Cas9 system for clinical purposes, some are using the tool to address fundamental questions about biology. Reuven Agami, a professor of genetics at the Netherlands Cancer Institute in Amsterdam, and his colleagues recently applied CRISPR to search for regulatory enhancer elements throughout the genome. They targeted the Cas9 nuclease to previously identified enhancer elements—transcription factor-binding DNA sequences that distally regulate gene expression—of two transcription factors: p53, and estrogen receptor alpha (ERα), which are both frequently mutated and deregulated in cancer. The team’s findings, published today (January 11) in Nature Biotechnology,highlight key enhancer sequences of these two proteins, and demonstrate the utility of CRISPR for the systematic study of noncoding DNA sequences. Two...
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We routinely say ‘Crispr’ing’ when we talk about changing specific genes – Paul Knoepfler, a biologist at the University of California

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A new gene-editing technology has been relentlessly hyped over the past year, and it goes by a catchy if rather incongruous acronym: Crispr. Hailed as the breakthrough of 2015 by Science Magazine, Crispr could potentially help eradicate diseases like malaria and, more controversially, could be used to modify the DNA of human embryos. But it’s safe to say that Crispr would not be grabbing the headlines if it were known by its full name: “clustered regularly-interspaced short palindromic repeats.” The acronym emerged out of email exchanges in 2001 between two microbiologists: Francisco Mojica of the University of Alicante in Spain, and Ruud Jansen, then at Utrecht University in the Netherlands. A team led by Dr. Jansen is believed to be...
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