Gene Editing News and Research

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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

Scientists develop human salivary gland organoids to test SARS-CoV-2 infectivity

In a recent study published in the journal Nature Cell Biology, researchers demonstrated the susceptibility of salivary glands to severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) infection in vitro using an organoid culture model.

20 Oct 2022

Novel method for gene editing of hPSCs yields up to 84% knockout efficiency

A Penn State-led team of interdisciplinary researchers has developed techniques to improve the efficiency of CRISPR-Cas9, the genome editing technique that earned the Nobel Prize in 2020.

9 Sep 2022

Study reveals the unique and unconventional way mosquitoes process odors

If you've ever sprayed yourself head to toe in bug repellent, yet still felt like a mosquito magnet, it will come as no shock to you that mosquitoes are very, very good at finding humans to bite.

19 Aug 2022

Next generation mRNA vaccines exhibit excellent protective and therapeutic effects

Researchers around the world have been working for years on developing vaccines against different types of cancer, but without much success. Now, Tufts School of Engineering researchers think they have found one that does work. They have devised a method of targeting cancer in mice with a vaccine that is so strong and precise, it eliminates tumors and even prevents their recurrence.

16 Aug 2022

Understanding the emotions behind memories

In this interview, we speak to Professor Kay Tye about her latest research into memories and how we can start to understand the emotions behind them.

10 Aug 2022

IRB scientists identify critical spots on the genome where gene editing could cause an unwanted response

CRISPR/Cas9 is a commonly used, very precise, gene editing technique whose development, by Jennifer A. Doudna and Emmanuelle Charpentier, was recognised with the 2020 Nobel Prize in Chemistry. Commonly known as "genetic scissors", CRISPR allows the introduction of the desired DNA sequence into (virtually) any spot of the genome, thus modifying or inactivating a gene. This technique is widely used in biomedical research and some CRISPR-based therapies are in clinical trials for the treatment of human blood disorders, some types of cancer and HIV, among other conditions.

10 Aug 2022

New dual-plasmid editing system for DNA-based information rewriting in vivo

DNA-based information is a new interdisciplinary field linking information technology and biotechnology.

5 Aug 2022

Turning to Organ-on-a-Chip Studies for the Future of Drug Discovery

In this interview, we speak to Tom Kostrzewski, VP of Science and Technology at CN Bio, about their organ-on-a-chip product and the future of drug discovery.

From CN-Bio 3 Aug 2022