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Gene therapy approach could help treat mitochondrial diseases

Researchers have developed a genome-editing tool for the potential treatment of mitochondrial diseases: serious and often fatal conditions which affect 1 in 5,000 people.

24 Sep 2018

Using CRISPR-Cas9 genome editing to correct mutation responsible for sickle cell disease

Genome editing using the CRISPR-Cas9 system has tremendous promise for therapeutic correction of genetic errors in human cells. Prior to adoption as a medical therapeutic, safety concerns relating to off-target effects must be minimized to mitigate risks that may arise from the unintended action of this system at sites similar, but not identical, to the desired gene target site.

From Integrated DNA Technologies 10 Aug 2018

New RNA deletion tool could help create drugs that correct genetic diseases

As scientists gain insights into which genes drive diseases, they are pursuing the next logical question: Can gene editing technologies be developed to treat or even cure those diseases? Much of that effort has focused on developing technologies such as CRISPR-Cas9, a protein-based system.

30 May 2018

LogicBio team to present data on preclinical effectiveness of GeneRide platform

LogicBio Therapeutics, Inc., a genetic medicine company founded to develop safe medicines with lasting therapeutic benefit for children with life-threatening diseases, today announced it will be presenting data on the company's technology and robust preclinical pipeline at the annual meeting of the American Society of Gene & Cell Therapy at the Hilton Chicago, from May 16-19, 2018.

14 May 2018

Scientists uncover new answers to cell aging in children with rare, fatal disease

In a recent paper published in Cell Reports, Saint Louis University researchers have uncovered new answers about why cells rapidly age in children with a rare and fatal disease.

20 Mar 2018

Benson Hill Biosystems granted patent for novel genome editing system

The U.S. Patent and Trademark Office has granted Benson Hill Biosystems Patent No. 9,896,696 related to CRISPR 3.0 Cms1 genome editing nucleases, further expanding the company's suite of genomics tools to accelerate crop performance improvements.

20 Feb 2018

Scientists develop ultrasound-propelled nanomotors for active delivery of Cas9-sgRNA complex

In cancer research, the “Cas-9–sgRNA” complex is an effective genomic editing tool, but its delivery across the cell membrane to the target (tumor) genome has not yet been satisfactorily solved. American and Danish scientists have now developed an active nanomotor for the efficient transport, delivery, and release of this gene scissoring system.

9 Feb 2018

Sangamo and Pfizer partner for development of new zinc finger protein gene therapy to treat ALS

Sangamo Therapeutics, Inc. and Pfizer Inc. today announced a collaboration for the development of a potential gene therapy using zinc finger protein transcription factors (ZFP-TFs) to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene.

8 Jan 2018

Study focuses on improving pharmacokinetic properties of therapeutic aptamers

Designing new therapeutic DNA aptamers with diverse side chains can improve their ability to interact with targets, and a new study describes characteristics of these side chains that may determine how long the aptamers remain in the bloodstream.

30 Nov 2017

Researchers visualize mechanism of CRISPR-Cas9 genetic-engineering technique

Researchers at Kanazawa University and the University of Tokyo report in Nature Communications the visualization of the dynamics of ‘molecular scissors’ — the main mechanism of the CRISPR-Cas9 genetic-engineering technique.

15 Nov 2017

UTSW receives $34 million CPRIT award for cancer research and faculty recruitment

The Cancer Prevention and Research Institute of Texas has awarded UT Southwestern researchers more than $34 million for cancer research and faculty recruitment, including support for programs in pancreatic cancer, lung cancer, breast cancer, and melanoma.

19 Sep 2017

IDT introduces first Cas9 enzyme variant that greatly reduces off-target effects in CRISPR genome editing

Integrated DNA Technologies today announced the launch of the first Cas9 enzyme variant that extensively reduces off-target effects in CRISPR genome editing without compromising on-target activity.

3 Aug 2017

Cellectis wins European patent for CRISPR use in T-cells

Cellectis, a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited CAR T-cells (UCART), today announced the grant by the European Patent Office of patent No. EP3004337 for the invention of using RNA-guided endonucleases, such as Cas9 or Cpf1 for the genetic engineering of T-cells.

24 Jul 2017

CRISPR may not be as foolproof as earlier believed – gene editing tech leads to unwanted mutations

CRISPR-Cas9 is a revolutionary new gene-editing technology that has been called “molecular scissors” and has been one of the handiest tools for genetic engineers in the recent times. It makes cutting and pasting gene sequences almost as easy as it sounds. CRISPR has been hailed as a breakthrough in genetic engineering.

30 May 2017

Study discovers new role for DNA2 enzyme in repair process

A biochemical analysis study has discovered a new role for the DNA2 enzyme.

6 Apr 2017

IDT offers cost-effective and most complete spectrum of CRISPR genome editing systems

Reflecting its drive to provide innovative solutions to the genomics community, Integrated DNA Technologies (IDT) is the first genomics company to develop and bring to market a complete ribonucleoprotein-based Cpf1 CRISPR system.

2 Feb 2017

Researchers uncover first ‘off-switches’ for better control of CRISPR/Cas9 gene editing

CRISPR/Cas9 genome editing is quickly revolutionizing biomedical research, but the new technology is not yet exact. The technique can inadvertently make excessive or unwanted changes in the genome and create off-target mutations, limiting safety and efficacy in therapeutic applications.

9 Dec 2016

Synthetic sgRNA for CRISPR genome editing

Chemically synthesized single guide RNA is the best way to do CRISPR genome editing. It is more pure than biological methods of creating guide RNAs, like in vitro transcription or plasmids. As a result, sgRNA leads to more consistency and higher quality.

8 Dec 2016

Okayama University researchers find way to prevent replication of influenza viral RNA

Researchers at Okayama University have successfully cleaved influenza viral RNA to prevent its replication using novel artificial RNA restriction enzymes in laboratory cell cultures.

30 Nov 2016

Scientists develop genetically engineered T cells with fusion inhibitor to disrupt HIV's harpoon

When HIV attacks a T cell, it attaches itself to the cell's surface and launches a "harpoon" to create an opening to enter and infect the cells.

18 Nov 2016