Gene Editing News and Research

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New genetic animal model may inform potential therapeutic options for Down syndrome

National Institutes of Health researchers compared a new genetic animal model of Down syndrome to the standard model and found the updated version to be more similar to the changes seen in humans.

14 Mar 2023

Common scientific method to analyze DNA in early human embryos fails to accurately reflect gene edits

A commonly used scientific method to analyze a tiny amount of DNA in early human embryos fails to accurately reflect gene edits, according to new research led by scientists at Oregon Health & Science University.

7 Mar 2023

OHSU researchers confirm first-ever nonhuman primate model of Usher syndrome

Those with Usher Syndrome -- the leading hereditary cause for simultaneous deafness and blindness, for which there is no treatment -- may have a new reason for hope now that researchers have confirmed the first-ever nonhuman primate model of their disease.

13 Feb 2023

Highly effective detection of cancer biomarkers using CRISPR

Most cancer diagnostic techniques rely on uncomfortable and invasive procedures, such as biopsies, endoscopies or mammograms.

1 Feb 2023

New discovery about microglia heralds myriad possibilities for treating neurodegenerative diseases

Combating Alzheimer's and other neurodegenerative diseases by inserting healthy new immune cells into the brain has taken a leap toward reality.

30 Jan 2023

AI tool promises to accelerate the development of gene therapies on a large scale

An artificial intelligence (AI) program may enable the first simple production of customizable proteins called zinc fingers to treat diseases by turning genes on and off.

26 Jan 2023

Protein discovery could lead to more effective treatments for lymphoma and lung cancer

Scientists from The Australian National University and the Peter MacCallum Cancer Centre have discovered that a protein, called Menin, contributes to abnormal deactivation of specific genes in cancer cells.

13 Jan 2023

Structure and function of a newly discovered CRISPR immune system revealed

In a rare feat, Utah State University biochemists Thomson Hallmark and Ryan Jackson, along with collaborators, published not one, but two seminal papers in a top-tier, peer-reviewed journal this week.

4 Jan 2023

Mutations responsible for common inherited heart condition corrected using CRISPR-Cas9 therapy

Using the CRISPR-Cas9 gene editing system, UT Southwestern researchers corrected mutations responsible for a common inherited heart condition called dilated cardiomyopathy (DCM) in human cells and a mouse model of the disease.

21 Dec 2022

Scientists discover new potential mechanism for vision loss

Visual cells in the human retina may not simply die in some diseases, but are mechanically transported out of the retina beforehand.

1 Dec 2022

New CRISPR-based tool can snip out faulty genes and replace them with new ones

Building on the CRISPR gene-editing system, MIT researchers have designed a new tool that can snip out faulty genes and replace them with new ones, in a safer and more efficient way.

24 Nov 2022

New family of adeno-associated viral vectors found to be better at delivering genetic cargo

Gene therapies can treat, even potentially cure, certain genetic diseases, but it is challenging to deliver the treatments to the parts of the body where they are needed.

22 Nov 2022

Discovery could lead to new mantle cell lymphoma drugs

A form of blood cancer known as mantle cell lymphoma is critically dependent on a protein that coordinates gene expression, such that blocking its activity with an experimental drug dramatically slows the growth of this lymphoma in preclinical tests, according to a study from Weill Cornell Medicine researchers.

22 Nov 2022

Gene mutation associated with autism causes an overstimulation of brain cells

Scientists looking to understand the fundamental brain mechanisms of autism spectrum disorder have found that a gene mutation known to be associated with the disorder causes an overstimulation of brain cells far greater than that seen in neuronal cells without the mutation.

21 Nov 2022

MIT researchers develop new technique for genome-wide studies of the liver

The liver’s ability to regenerate itself is legendary. Even if more than 70 percent of the organ is removed, the remaining tissue can regrow an entire new liver.

21 Nov 2022

What are the current challenges and future directions of mRNA nanomedicine?

Researchers presented challenges related to the clinical translation of messenger ribonucleic acid (mRNA) technology, recent innovations and future directions to improve the clinical efficacy of mRNA nanomedicine.

15 Nov 2022

Novel gene editing therapy reduces circulating TTR protein levels in patients with ATTR amyloid cardiomyopathy

A single IV infusion of NTLA-2001, a novel CRISPR/Cas9-based gene editing therapy, significantly reduced circulating transthyretin (TTR) protein levels in patients with ATTR amyloid cardiomyopathy, a progressive and fatal cause of heart failure, according to late-breaking research presented today at the American Heart Association’s Scientific Sessions 2022.

7 Nov 2022

Beyond vaccines: emerging technologies capable of unlocking the promise of biologically targeted mRNA therapeutics

Researchers reviewed technology advancements that will unlock the promise of biologically targeted messenger ribonucleic acid (mRNA) therapeutics beyond vaccines.

7 Nov 2022

New control system could be used to finely tune the production of useful proteins

Using an approach based on CRISPR proteins, MIT researchers have developed a new way to precisely control the amount of a particular protein that is produced in mammalian cells.

1 Nov 2022

New studies point to a broadly applicable treatment for neurodegenerative diseases

Two new studies from Washington University School of Medicine in St. Louis support development of a broadly applicable treatment for neurodegenerative diseases that targets a molecule that serves as the central executioner in the death of axons, the wiring of the nervous system.

26 Oct 2022