Gene Editing News and Research

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Changes in cellular metabolites regulate earliest stages of embryonic stem cell development

Changes in cellular metabolites have been shown to regulate embryonic stem cell development at the earliest stages of life. Metabolites are simple compounds generated during life-sustaining chemical activities in cells.

17 Nov 2015

Boston Children's Hospital selects Vijay G. Sankaran to receive Rising Star Award

Saluting his spectacular track record to date of innovative research on red blood cell disorders and his future promise as a physician/scientist, Boston Children's Hospital has selected Vijay G. Sankaran, MD, PhD, of Dana-Farber/Boston Children's Cancer and Blood Disorders Center to receive the Rising Star Award at the hospital's third annual Global Pediatric Innovation Summit, Taking on Tomorrow (#PedInno15).

11 Nov 2015

Stem cell biology and gene-editing techniques offer hope for kidney regeneration

Mini-kidney organoids have now been grown in a laboratory by using genome editing to re-create human kidney disease in petri dishes.

23 Oct 2015

DNA study reveals basic principle of gene regulation

A study of where and how an enzyme cuts DNA may have inadvertently revealed a basic principle of gene regulation, say researchers in Boston Children's Hospital's Program in Cellular and Molecular Medicine (PCMM).

23 Oct 2015

Using deep learning to analyze genetic mutations: an interview with Brendan Frey

To understand deep learning in the context of genetic disease, you need to understand shallow learning first. Shallow learning relates mutations to diseases by looking for mutations that commonly occur in patients with a disease. It’s a commonly used method.

21 Sep 2015

New collaboration may allow researchers to develop potential gene therapies for pediatric diseases

A new collaboration between Seattle Children's Research Institute and bluebird bio, Inc., a biotechnology company based in Cambridge, Mass. and Seattle, will allow researchers to develop potentially transformative gene therapies for severe genetic and rare pediatric diseases. The collaboration supports pediatric researchers in Seattle who will work with bluebird bio scientists and the company's gene editing technology to research potential cures for genetic pediatric diseases.

11 Sep 2015

CRISPR tool streamlines gene editing

Within the past few years, a new technology has made altering genes in plants and animals much easier than before. The tool, called CRISPR/Cas9 or just CRISPR, has spurred a flurry of research that could one day lead to hardier crops and livestock, as well as innovative biomedicines. But along with potential benefits, it raises red flags, according to an article in Chemical & Engineering News (C&EN), the weekly newsmagazine of the American Chemical Society.

10 Sep 2015

UCSF study reveals molecular timing mechanism of stem cells

UC San Francisco researchers have for the first time developed a method to precisely control embryonic stem cell differentiation with beams of light, enabling them to be transformed into neurons in response to a precise external cue.

27 Aug 2015

New technology advances genome engineering

In his mind, Basil Hubbard can already picture a new world of therapeutic treatments for millions of patients just over the horizon. It's a future in which diseases like muscular dystrophy, cystic fibrosis and many others are treated permanently through the science of genome engineering.

11 Aug 2015

Deerfield provides $20 million financing to Editas Medicine

Deerfield Management Company, L.P.'s venture capital fund, Deerfield Healthcare Innovations Fund, L.P., today announced its first therapeutics-based investment by participating, along with Deerfield Private Design Fund III, in providing financing to Editas Medicine. In total, Deerfield invested $20 million in this early-stage institutional financing.

10 Aug 2015

New synthetic gene drives could one day improve human health and the environment

Gene drives are genetic elements - found naturally in the genomes of most of the world's organisms - that increase the chance of the gene they carry being passed on to all offspring, and thus, they can quickly spread through populations. Looking to these natural systems, researchers around the world, including some Wyss Institute scientists, are developing synthetic gene drives that could one day be leveraged by humans to purposefully alter the traits of wild populations of organisms to prevent disease transmission and eradicate invasive species.

31 Jul 2015

Deep Genomics set to bring the power of deep learning technologies to genomics

Evolution has altered the human genome over hundreds of thousands of years -- and now humans can do it in a matter of months.

23 Jul 2015

Researchers use gene-editing technique involving low-dose irradiation to repair human stem cells

For the first time, researchers have employed a gene-editing technique involving low-dose irradiation to repair patient cells, according to a study published in the journal Stem Cells Translational Medicine. This method, developed by researchers in the Cedars-Sinai Board of Governors Regenerative Medicine Institute, is 10 times more effective than techniques currently in use.

23 Jul 2015

Study opens new area of discovery for different aspects of cell biology, biomedical research

Every cell in the body uses phosphorylation, the process of adding a chemical tag to control a protein's function and fate, such as when it moves from one part of a cell to another or binds to other proteins.

19 Jun 2015

EU research project to study how social inequalities influence healthy ageing

The Lifepath project, involving 15 institutions in Europe, the US and Australia, has been supported by a six million euro grant from the EU's Horizon 2020 programme...

10 Jun 2015

New UAB research identifies link between temporal lobe epilepsy and memory loss

New research from the University of Alabama at Birmingham identifies an epigenetic cause for why patients with temporal lobe epilepsy tend to have memory loss, and suggests a potential way to reverse that loss.

8 Jun 2015

NAS, NAM launch major initiative to guide decision making on human gene-editing research

The National Academy of Sciences and the National Academy of Medicine are launching a major initiative to guide decision making about controversial new research involving human gene editing. Human gene-editing technologies, such as CRISPR-Cas9, may lead to promising new treatments for disease.

19 May 2015

Salk discovery may offer new avenues for generating novel therapies

Scientists at the Salk Institute have discovered a novel type of pluripotent stem cell--cells capable of developing into any type of tissue--whose identity is tied to their location in a developing embryo. This contrasts with stem cells traditionally used in scientific study, which are characterized by their time-related stage of development.

7 May 2015

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Sangamo BioSciences, Inc. announced that data from clinical, preclinical and research-stage programs focused on the development of ZFP Therapeutics will be presented at the 18th Annual Meeting of the American Society of Gene and Cell Therapy

29 Apr 2015

Yale researchers successfully correct gene mutation that causes cystic fibrosis

Yale researchers successfully corrected the most common mutation in the gene that causes cystic fibrosis, a lethal genetic disorder. The study was published April 27 in Nature Communications.

28 Apr 2015