Sentences with phrase «crispr nuclease»

Announced a worldwide collaboration with Sangamo Therapeutics, Inc. (Sangamo) using Sangamo's zinc finger nuclease technology platform for the development of next - generation ex vivo cell therapies in oncology.

These risks and uncertainties include: Gilead's ability to achieve its anticipated full year 2018 financial results; Gilead's ability to sustain growth in revenues for its antiviral and other programs; the risk that private and public payers may be reluctant to provide, or continue to provide, coverage or reimbursement for new products, including Vosevi, Yescarta, Epclusa, Harvoni, Genvoya, Odefsey, Descovy, Biktarvy and Vemlidy ®; austerity measures in European countries that may increase the amount of discount required on Gilead's products; an increase in discounts, chargebacks and rebates due to ongoing contracts and future negotiations with commercial and government payers; a larger than anticipated shift in payer mix to more highly discounted payer segments and geographic regions and decreases in treatment duration; availability of funding for state AIDS Drug Assistance Programs (ADAPs); continued fluctuations in ADAP purchases driven by federal and state grant cycles which may not mirror patient demand and may cause fluctuations in Gilead's earnings; market share and price erosion caused by the introduction of generic versions of Viread and Truvada, an uncertain global macroeconomic environment; and potential amendments to the Affordable Care Act or other government action that could have the effect of lowering prices or reducing the number of insured patients; the possibility of unfavorable results from clinical trials involving investigational compounds; Gilead's ability to initiate clinical trials in its currently anticipated timeframes; the levels of inventory held by wholesalers and retailers which may cause fluctuations in Gilead's earnings; Kite's ability to develop and commercialize cell therapies utilizing the zinc finger nuclease technology platform and realize the benefits of the Sangamo partnership; Gilead's ability to submit new drug applications for new product candidates in the timelines currently anticipated; Gilead's ability to receive regulatory approvals in a timely manner or at all, for new and current products, including Biktarvy; Gilead's ability to successfully commercialize its products, including Biktarvy; the risk that physicians and patients may not see advantages of these products over other therapies and may therefore be reluctant to prescribe the products; Gilead's ability to successfully develop its hematology / oncology and inflammation / respiratory programs; safety and efficacy data from clinical studies may not warrant further development of Gilead's product candidates, including GS - 9620 and Yescarta in combination with Pfizer's utomilumab; Gilead's ability to pay dividends or complete its share repurchase program due to changes in its stock price, corporate or other market conditions; fluctuations in the foreign exchange rate of the U.S. dollar that may cause an unfavorable foreign currency exchange impact on Gilead's future revenues and pre-tax earnings; and other risks identified from time to time in Gilead's reports filed with the U.S. Securities and Exchange Commission (the SEC).

In recent years several techniques, such as CRISPR / Cas9 or zinc finger nucleases have been experimented to directly modify the DNA of plants and animals.

«CRISPR has proven so easy and inexpensive that Dana Carroll of the University of Utah, Salt Lake City, who spearheaded the development of zinc finger nucleases, [one of its competitors,] says it has brought about the «democratization of gene targeting.

By using engineered zinc - finger nucleases (ZFNs) designed to target an integrated reporter and two endogenous rat genes, Immunoglobulin M (IgM) and Rab38, we demonstrate that a single injection of DNA or messenger RNA encoding ZFNs into the one - cell rat embryo leads to a high frequency of animals carrying 25 to 100 % disruption at the target locus.

Scientists can select from a variety of scalpels, including zinc finger nucleases, TALENs and CRISPR / Cas9.

In plants and Caenorhabditis elegans, two distinct populations of small RNAs have been proposed to participate in RNAi: «Primary siRNAs» (derived from DICER nuclease - mediated cleavage of the original trigger) and «secondary siRNAs» [additional small RNAs whose synthesis requires an RNA - directed RNA polymerase (RdRP)-RSB-.

Nuclease and chemical protection studies with the 52mer helped to define the DNA base pairs that contributed to the specificity of binding.

GUIDE - seq enables genome - wide profiling of off - target cleavage by CRISPR - Cas nucleases.

Development and evaluation of a fluorogenic 5 ′ nuclease assay to detect and differentiate between Ebola virus subtypes Zaire and Sudan

«Before CRISPR, there was TALENS [transcription activator - like effector nucleases] and zinc finger nucleases — older technologies that were not as precise or reliable,» explains Sosa.

Talk of curing AIDS made front - page news last year, in part due to an astonishing new gene - editing technology: lab - engineered proteins called zinc finger nucleases.

To go further in the TREX2 role in homeostasis and skin pathogenesis, the researchers wanted to see the role of this nuclease on psoriasis.

And in another advance, virologist Paula Cannon of the University of Southern California used zinc finger nucleases to create human stem cells that lack CCR5.

In that case, six patients suspended antiretroviral therapy for 12 weeks after infusion with zinc finger nuclease — altered CD4 cells.

KIM Jin - soo, Director of the IBS Center for Genome Editing and corresponding author of the two studies commented, «Since the two studies have proved the superior specificity of Cpf1, this new nuclease will be more widely used for precise genome editing that does not produce any unintended mutations.

Ultimately, this week's discourse will lead to a consensus statement providing some guidance on how to approach using this and older gene editing technologies such as zinc finger nucleases and enzymes called transcription activator - like effector nucleases, or TALENs.

They digested human genomic DNA using Cas9 nucleases in a test tube, which was then subjected by whole genome sequencing.

Furthermore, by adding guanine nucleotides at the end of sgRNA (single guided RNA) that composes CRISPR - Cas9, they have successfully created this highly - developed programmable nuclease, which has no measurable off - target effects in the human genome.

Staplhylococcal nuclease undergoes a reversible structural transition between ph3 and 4 which be mesured by changes in tryptoham fluorescence.

Its competitors — designer proteins called zinc finger nucleases and TALENs — also precisely alter chosen DNA sequences, and several companies are already exploiting them for therapeutic purposes in clinical trials.

But CRISPR has proven so easy and inexpensive that Dana Carroll of the University of Utah, Salt Lake City, who spearheaded the development of zinc finger nucleases, says it has brought about the «democratization of gene targeting.»

They have used a different technique, called zinc finger nucleases, to disrupt a gene on T cells that HIV uses to enter the cells.

The viral scraps serve as an infection memory bank: From them, bacteria create guide RNAs that can seek out the DNA of returning viruses before chopping up the viral genes with a nuclease.

A stopped - flow spectrofluorometer was used to study the kinetics renaturation of nuclease from the acidified form on neutralization, the refolding is fast and the data can be described as a sequence of two first - order processes with half times of about 55 and 350 milliseconds, respectively.

Although the study looked at CRISPR - Cas9 gene editing, the researchers believe their findings extend to other gene - editing tools such as zinc - finger nucleases (ZFN) and TAL effector nucleases.

One concern, for example, is that the nucleases could cause mutations at locations other than those targeted.

In clinical trials already underway, for example, researchers have used an older gene - editing technique, enzymes call zinc finger nucleases, in immune cells to deactivate the gene for CCR5, a surface protein that HIV latches onto in order to infect cells.

Shown to work just 3 years ago, CRISPR consists of a an enzyme called a nuclease and a piece of RNA that homes in on a targeted DNA sequence, enabling the enzyme to introduce precisely targeted mutations, corrections to mutations, or other alterations.

Just this week, for example, a team led by Feng Zhang of the Broad Institute of Harvard and MIT, one of the pioneers of the method, published a paper in Science on engineering the nuclease part of CRISPR so that it more accurately cuts the intended DNA target.

These techniques use enzymes called nucleases to snip DNA at specific points and then delete or rewrite the genetic information at those locations.

Meanwhile, Cellectis announced it now has «an umbrella patent» that its CEO, Andre Choulika, says «covers most of the gene editing procedures done with a nuclease,» including those based on CRISPR - Cas 9, TALENs, zinc fingers, and many meganucleases.

Moreover, they can be degraded quickly by special enzymes (nucleases) that are present in bodily fluids such as saliva or blood that digest foreign DNA.

A similar mutation, detected by S1 nuclease mapping of LDL receptor messenger RNA, occurred in a patient with familial hypercholesterolemia whose receptor also fails to be transported to the cell surface.

In 2009, for example, Sangamo Therapeutics in Richmond, California, began using zinc finger nucleases to modify genes in immune cells from HIV - infected people, hoping to make the cells resistant to the virus.

Thus far, the connection between the gene, which produces a DNA - cutting enzyme called a nuclease, and the kidney disease has remained obscure.

«We don't know what it is seeing that other nucleases aren't seeing.»

Conventional CRISPR uses a guide RNA (gRNA) coupled with an enzyme known as a nuclease, most commonly Cas9, that together attach to a specific stretch of DNA bases; the nuclease then snips the double helix.

«If the probe gets cleaved by serum nucleases, then our probe would be lit up all over the bloodstream.

In 2009, researchers at Dow AgroSciences in Indianapolis, Indiana, and Sangamo BioSciences in Richmond, California, announced that they had used enzymes called zinc - finger nucleases to insert a gene for herbicide resistance at a specific site in the maize genome (V. K. Shukla et al..

At sites complementary to the crRNA - guide sequence, the Cas9 HNH nuclease domain cleaves the complementary strand, whereas the Cas9 RuvC - like domain cleaves the noncomplementary strand.

«Gene editing based on nucleases is very good at inactivating genes,» says CRISPR researcher Feng Zhang of the Broad Institute in Cambridge, Massachusetts.

The urinary cells were collected from patients with the chromosomal inversions causing hemophilia to make iPSCs, the team applied CRISPR - Cas9 nucleases (Clustered Regularly Interspaced Short Palindromic Repeats - CRISPR associated protein 9) to them.

What's more, say the researchers, the cancer - causing effects of off - target deletions mistakenly created by the V (D) J enzyme need to be considered in designing site - specific enzymes for genome modification such as zinc - finger nucleases, TALENS, or CRISPRs.

Base editors borrow from CRISPR's components — guide RNAs (gRNAs) and Cas9 or other nucleases — but don't cut the double helix and instead chemically alter single bases with deaminase enzymes such as TadA and ADAR.

In tests, nucleases (or enzymes) produced by the staph bacteria cleave the particles, like a warrior wielding a sword.

According to Director Jin - Soo Kim, «We used CRISPR RGENs [RNA - guided engineered nucleases] to repair two recurrent, large chromosomal inversions responsible for almost half of all severe hemophilia A cases.»

McNamara acknowledges previous research by Arthur Arnone, UI professor emeritus in biochemistry, who was the first to define the structure of the S. aureus nuclease.

But since it's split only by staph nucleases, then we can pinpoint where the staph bacteria are active.»

Just as important, the UI probe has been chemically modified so that it's shredded only by the staph bacteria's nuclease and not by a nuclease secreted by normal, healthy cells.