Gene Editing News and Research

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Research suggests an avenue to treat, prevent genetic disorders in the womb

New research led by hearing scientists at Oregon Health & Science University suggests an avenue to treat and prevent intractable genetic disorders before birth.

6 Apr 2020

Horizon Discovery expands cell-based CRISPR screening services

Horizon Discovery Group plc, today announced the addition of an arrayed CRISPR knockout screening service for primary human B cells.

From Revvity 6 Apr 2020

Scientists examine the molecular causes of rare hereditary disease

Scientists in EMBL Barcelona’s Ebisuya group, with collaborators from RIKEN, Kyoto University, and Meijo Hospital in Nagoya, Japan, have studied oscillating patterns of gene expression, coordinated across time and space within a tissue grown in vitro, to explore the molecular causes of a rare human hereditary disease known as spondylocostal dysostosis.

5 Apr 2020

Horizon Discovery and Pharmahungary report positive early-stage results for novel micro-RNA therapeutic for ischemic heart diseases

Horizon Discovery Group plc and Pharmahungary Group announced positive early-stage results for a novel micro-RNA therapeutic for ischemic heart diseases.

31 Mar 2020

New method to treat blindness using retinal cell production

Scientists in Sweden have innovated a method of treating blindness using embryonic stem cells to produce retinal cells.

30 Mar 2020

Researchers discover new way to refine production of retinal cells to treat blindness

Researchers at Karolinska Institutet and St Erik Eye Hospital in Sweden have discovered a way to refine the production of retinal cells from embryonic stem cells for treating blindness in the elderly.

30 Mar 2020

Finding new treatments for muscular dystrophy with CRISPR-Cas9

A new study out of Boston's Children's Hospital has used the gene-editing tool CRISPR-Cas9 to explore the fatal genetic condition called facioscapulohumeral dystrophy (FSHD, one of the family of muscular dystrophies), and to test out the potential utility of various genes involved in this disorder. The research is published in the journal Science Translational Medicine.

25 Mar 2020

Humans are not the first to exploit benefits of CRISPR technique, research shows

In recent years, the development of CRISPR technologies and gene-editing scissors in particular have taken the world by storm. Indeed, scientists have learned how to harness these clever natural systems in the biotech and pharmaceutical industries, among other areas.

24 Mar 2020

Blood stem cells conserve epigenetic memory of previous infections to boost immunity

These findings should have a significant impact on future vaccination strategies and pave the way for new treatments of an underperforming or over-reacting immune system. The results of this research are published in Cell Stem Cell on March 12, 2020.

13 Mar 2020

Fight for Sight funded researcher develops gene therapy approach for glaucoma

A Fight for Sight funded researcher from the University of Bristol has used gene editing techniques to successfully demonstrate a gene therapy for glaucoma in the lab, in results published in Molecular Therapy in March.

11 Mar 2020

Study reports second ever case of a patient to be cured of HIV

A study of the second HIV patient to undergo successful stem cell transplantation from donors with a HIV-resistant gene, finds that there was no active viral infection in the patient's blood 30 months after they stopped anti-retroviral therapy, according to a case report published in The Lancet HIV journal and presented at CROI.

10 Mar 2020

Researchers identify a gut microbe generated byproduct linked to cardiovascular disease

Cleveland Clinic researchers have identified a gut microbe generated byproduct - phenylacetylglutamine - that is linked to development of cardiovascular disease, including heart attack, stroke and death.

5 Mar 2020

Preclinical data of Ocugen's OCU400 genetic modifier published in Nature Gene Therapy

Ocugen, Inc., a clinical-stage company focused on discovering, developing and commercializing transformative therapies to treat rare and underserved ophthalmic diseases, announced today the publication in Nature Gene Therapy of preclinical data of nuclear hormone receptor gene NR2E3 as a genetic modifier and therapeutic agent to treat multiple retinal degenerative diseases.

3 Mar 2020

Gene-editing technology may lead to new model for studying Parkinson’s disease

Parkinson's disease researchers have used gene-editing tools to introduce the disorder's most common genetic mutation into marmoset monkey stem cells and to successfully tamp down cellular chemistry that often goes awry in Parkinson's patients.

27 Feb 2020

New form of gene editing slows ALS progression in mice

With a new CRISPR gene-editing methodology, scientists from the University of Illinois at Urbana-Champaign inactivated one of the genes responsible for an inherited form of amyotrophic lateral sclerosis - a debilitating and fatal neurological disease for which there is no cure.

26 Feb 2020

Gene-editing is more error-prone than thought, new findings suggest

The standard gene-editing tool, CRISPR-Cas9, frequently produces a type of DNA mutation that ordinary genetic analysis misses, claims new research published in the journal Proceedings of the National Academy of Sciences.

26 Feb 2020

Scientists discover new biological mechanism of insulin signaling

In a discovery that may further the understanding of diabetes and human longevity, scientists at Scripps Research have found a new biological mechanism of insulin signaling.

25 Feb 2020

New biological tool can detect and debug faulty genetic circuits

Every cell in our body has a computer-like control system that sends biological signals through thousands of circuits to monitor the cell's needs and regulate its responses.

16 Feb 2020

Minigenes produced by CRISPR combat liver disease in mice

A new gene-editing technique based on CRISPR was used to successfully avoid the development of a liver disease that can be caused by hundreds of mutations, as well as to ease the symptoms in mice. This proof-of-concept study was published online in the journal Science Advances in February 2020.

12 Feb 2020

Newly discovered large phages blur the life/non-life boundary

A new study published in the journal Nature shows that there are literally hundreds of viruses large enough to consume bacteria, and with properties that are typical of a living organism rather than the non-living self-replicating packages of DNA/RNA that viruses are often assumed to be.

12 Feb 2020