Sentences with phrase «nucleases for»

His work also contributed to advance the use of artificial nucleases for targeted genome editing in cell and gene therapy.

Compounding this risk, the liver cells that now contain the DNA for ZFNs will keep making the nucleases for perhaps years, even though they are no longer needed to guide the new gene to its spot in the genome.

Not exact matches

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 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.

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.

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.

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.

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.

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..

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.

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.»

In it, they sound the alarm about new genome - editing techniques known as CRISPR and zinc - finger nucleases that make it much easier for scientists to delete, add, or change specific genes.

The class 2 systems have evolved from class 1 systems via the insertion of transposable elements encoding various nucleases, and are now being used as tools for genome editing.

For humanized mice samples, whole genome amplification using the REPLI - g Mini Kit (Qiagen) was conducted prior to the surveyor nuclease assay due to limiting cell numbers.

Cell growth was monitored every 48 hours post-stimulation for approximately two weeks and the efficiency of CXCR4 disruption was assessed at day five post-transduction by both the Surveyor nuclease assay and by deep - sequencing of the CXCR4 target site.

The 50 uL PCR reaction consisted of the following mixture: 10 uL 5X LongAmp Taq Reaction Buffer, 1.5 uL of dNTPs (1 mM), 4 uL of each primer pairs (10 uM each, see below for details), 2 uL of template DNA, 2 uL of LongAmp Hot Start Taq DNA polymerase (2500 U / mL) and 30.5 uL of nuclease - free water.

A similar approach, but using a different technology (zinc finger nucleases), was reported for Huntington's disease in 2012.

Cas genes, which code for polymerases, nucleases, and helicases, were also disrupted to elucidate their role in the process.

A metagenomics analysis finds Cas9 in archaea for the first time, along with two previously unknown Cas nucleases from bacteria.

Engineered CRISPR systems for gene editing now contain two main components, a single guide RNA or sgRNA and Cas - 9 nuclease.

Science magazine heralded these transcription activator - like effector nuclease proteins, known as TALENS, as a major scientific breakthrough in 2012, nicknaming them «genomic cruise missiles» for their ability to allow researchers to target specific locations with great efficiency.

It was suspected, but not known for certain, that fly as well as mouse Zucchini proteins were nucleases — enzymes that specialize in cleaving bonds between the nucleotide subunits of nucleic acids (DNA and RNA).

The variety of new tools available for genetic manipulation now include lentiviral - based gene delivery, and gene editing using CRISPR / Cas9, zinc finger nucleases (ZFNs) or transcription activator - like effector nucleases (TALENs).

This protocol describes a system for efficient genome editing in hPSCs using engineered transcription activator - like effector nucleases (TALENs) or clustered regularly interspaced short palindromic repeat (CRISPR) technology.

Please note that this off - target score is available for human genome with SpCas9 nucleases.

Cas9 nucleases from Streptococcus pyogenes (SpCas9) have been widely used for both gene knockout and knock - in at the level of single or multiple genes.

A while later, in 2002, scientists spotted that there are DNA instructions for making a nuclease very near these repeats.

software for analysis of PGM sequencing of DNA targeted with artificial sequence - specific nucleases

The use of viral vector for mouse mutagenesis and / or for delivery of gene editing by nuclease (F. Piguet, IGBMC)

The nucleases were capable of pushing out an undesirable trait in a plant — such as a gene for a chemical that acted as an environmental pollutant when consumed by livestock — and replacing it with a desirable trait, such as herbicide resistance, in a single shot.

Eventually, the team of Dow and Sangamo scientists showed that it could engineer zinc finger nucleases that gave a two - for - one deal.

These systems, however, require efficient design and time - consuming assembly of nuclease constructs for DNA targeting.

The enzyme activities of Caf1 and Ccr4 are both required for deadenylation by the human Ccr4 — Not nuclease module

A patent application has been filed and testing is underway on the protein — called HT - TALENs (short for HIV - targeted transcription activator - like effector nucleases)-- which uses a newly developed gene - editing technique to rid the body's cells of the immunodeficiency virus before it has a chance to multiply and possibly develop into AIDS.

This toolkit includes TALEN and CRISPR nucleases, nickases, and gene activation systems as well as Cys4, tRNA, and ribozyme systems for multiplex gRNA expression.