Genome Editing News and Research

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UB research identifies key mechanism in infantile cystinosis

University at Buffalo research has identified how a misstep in the genesis of a key component of the kidney causes infantile cystinosis, a rare disease that significantly shortens the lifespan of patients.

11 Dec 2023

New map offers valuable insights into the underlying mechanisms of neurodevelopmental disorders and cancer

Scientists have created the first extensive map showing how all possible genetic changes can affect health in the gene DDX3X, leading to valuable insights into the underlying mechanisms of neurodevelopmental disorders and cancer.

11 Dec 2023

CRISPR-based gene-editing therapy could offer hope for people with inherited high LDL cholesterol

A single infusion of a CRISPR-based gene-editing therapy significantly reduced low-density lipoprotein cholesterol (LDL, the "bad cholesterol") in people who carry one gene for the inherited condition that results in very high LDL cholesterol levels and a high risk of heart attack at an early age, according to late-breaking science presented today at the American Heart Association's Scientific Sessions 2023.

13 Nov 2023

Science, Innovation, and Xenotransplantation: A Discussion with Dr. Wenning Qin

In this interview, we delve deep into the pioneering world of xenotransplantation and the evolving landscape of gene editing.

2 Nov 2023

Proof-of-concept study holds promise for safer gene therapy techniques for treating immunodeficiencies

Researchers described an approach to edit the recombination-activating gene 2 (RAG2) locus for therapeutic applications.

1 Nov 2023

Novel replacement strategy holds promise for the treatment of various genetic disorders

Severe Combined Immunodeficiencies (SCIDs) are a group of debilitating primary immunodeficiency disorders, primarily caused by genetic mutations that disrupt T-cell development. SCID can also affect B-cell and natural killer cell function and counts.

27 Oct 2023

Is retroelement-based gene editing a safer alternative to CRISPR-Cas approaches?

In a Perspective, Stephen Tang and Samuel Sternberg discuss retroelement-based gene editing as a safer alternative to CRISPR-Cas approaches.

26 Oct 2023

New software tool provides a way for safer design of genome editing

A team of researchers has developed a software tool called DANGER (Deleterious and ANticipatable Guides Evaluated by RNA-sequencing) analysis that provides a way for the safer design of genome editing in all organisms with a transcriptome.

24 Oct 2023

Gene therapy breakthrough: Scientists develop method to deliver larger genes to target cells

Gene therapy currently represents the most promising approach for the treatment of hereditary diseases. Yet despite significant breakthroughs in recent years, there are still a number of hurdles that hinder the wider application of gene therapies.

24 Oct 2023

Plant geneticists explore predictability of natural and CRISPR mutations

For tens of thousands of years, evolution shaped tomatoes through natural mutations. Then, humans came along. For centuries, we've bred and cherry-picked tomatoes with our preferred traits.

19 Oct 2023

Genetically edited chickens show resistance to avian influenza in landmark study

Researchers use CRISPR technology to create chickens resistant to avian influenza, targeting specific genes to inhibit viral replication. The study reveals both the promise and complexities of using genetic editing as a disease control strategy, also shedding light on influenza's adaptability.

11 Oct 2023

Researchers spearhead a new method to hone in on new cancer drug targets

Searching for new ways to block the growth of cancer cells is like looking for a needle in a haystack. Tumor cells rely on thousands of proteins to function, but only a few of those proteins can be precisely targeted by drugs to treat cancer safely and effectively.

5 Oct 2023

Sphere Fluidics’ Cyto-Mine system selected by FairJourney Biologics to advance cell line development workflows

Sphere Fluidics, a company developing single cell analysis systems underpinned by its patented picodroplet technology, today announced the Cyto-Mine® platform has been selected by FairJourney Biologics, a world leading end-to-end antibody partner, providing integrated services across antibody discovery, engineering, and production, to expand its cell line development capabilities.

From Fluidic Sciences and Sphere Bio 4 Oct 2023

NIH grant to fund development of new gene therapy platform for brain diseases

A two-phase NIH grant will fund research into a new CRISPR-based gene therapy platform that will target genetic brain diseases like Angelman syndrome and H1-4 (HIST1H1E) syndrome.

3 Oct 2023

New CRISPR-based gene-editing tool could lead to better treatments for genetic disorders

A new CRISPR-based gene-editing tool has been developed which could lead to better treatments for patients with genetic disorders.

29 Sep 2023

Arrayed CRISPR Screening for Revolutionizing Target Discovery

In this interview conducted at SLAS EU 2023 in Brussels, Belgium, we spoke to Ulrike Kuenzel, Associate Director in the Functional Genomics department at AstraZeneca about the application of arrayed CRISPR screening for target identification and more.

From Society for Laboratory Automation and Screening (SLAS) 27 Sep 2023

Study reveals non-coding genetic variants associated with Alzheimer’s disease functioning in microglia

Scientists studying Alzheimer's disease (AD) have identified thousands of genetic variants in the genome in the development of this progressive neurodegenerative disease.

23 Sep 2023

Scientists discover novel microglia-associated risk factors for Alzheimer's disease

Scientists studying Alzheimer's disease (AD) have identified thousands of genetic variants in the genome in the development of this progressive neurodegenerative disease.

22 Sep 2023

Rice engineers develop split gene-editing tool with enhanced control

To make a gene-editing tool more precise and easier to control, Rice University engineers split it into two pieces that only come back together when a third small molecule is added.

21 Sep 2023

Scientists develop Australia’s first-of-its-kind next-gen mpox diagnostic tool

In a collaborative study published in The Lancet Microbe, the team of scientists, led by the Peter Doherty Institute for Infection and Immunity (Doherty Institute) and WEHI (Walter and Eliza Hall Institute of Medical Research), revealed MPXV-CRISPR – a powerful diagnostic tool capable of detecting MPXV in clinical samples with acute precision and at a faster rate than any other method, thanks to the power of CRISPR technology.

20 Sep 2023