Gene Editing News and Research

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Gene editing breakthrough could offer new hope for people with phenylketonuria

Phenylketonuria (PKU) is a rare newborn genetic disease that impacts between 1 in 10,000 to 1 in 20,000 people, depending on the individuals' genetic ancestry. PKU causes an amino acid-;called phenylalanine (Phe)-;to build up in the bloodstream.

2 Nov 2023

Loss of structural protein ACTL7B blocks spermatogenesis in male mice

Mature spermatozoa are characterized by an head, midpiece and a long tail for locomotion. Now, researchers from the University Hospital Bonn (UKB) and the Transdisciplinary Research Unit "Life & Health" at the University of Bonn have found that a loss of the structural protein ACTL7B blocks spermatogenesis in male mice.

27 Oct 2023

Strengthening the immune system to fight cytomegalovirus on its own

Some viruses can be dormant throughout a person's life and cause no harm but become dangerous when the immune system is weakened.

17 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

Broken String Biosciences strengthens senior leadership team to accelerate product development and commercialization

Broken String Biosciences, a genomics company building a technology platform to drive the development of cell and gene therapies that are safer by design, today announced the expansion of its senior leadership team.

From Broken String Biosciences 2 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

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

Study reports an increase in incidence of islet autoimmunity after SARS-CoV-2 infection in young children

Type 1 diabetes is an autoimmune disease leading to an impaired glucose metabolism and requires life-long administration of insulin.

8 Sep 2023

Scientists discover new role for liver tPA in regulating cholesterol levels

Despite advances in treatment for high cholesterol, heart disease remains the leading cause of death in the U.S. Scientists at the Medical College of Wisconsin (MCW) are investigating the role of a form of cholesterol called very-low-density lipoprotein – and their findings may lead to new treatment options in the future.

1 Sep 2023

Scientists delve deeper into how vaccines work against leishmaniasis

Scientists have established the effectiveness of vaccines they developed to prevent the disfiguring skin disease leishmaniasis in animal studies, and Phase 1 human trial planning is in motion for the most promising candidate.

31 Aug 2023

UC researchers study parasite that causes Chagas disease

Almost everything about insects called kissing bugs is revolting, from the insidious way they bite people's faces at night to drink their blood while they sleep to the way they spread disease through their poop.

23 Aug 2023

Gene therapy repairs neural connections in mice with Hurler syndrome

A new study from the University of Minnesota is the first to demonstrate the ability for gene therapy to repair neural connections for those with the rare genetic brain disorder known as Hurler syndrome.

21 Aug 2023

New gene-editing tool helps to study single-base mutations in cancer

A change in just one letter in the code that makes up a cancer-causing gene can significantly affect how aggressive a tumor is or how well a patient with cancer responds to a particular therapy.

10 Aug 2023

Can genome editing transform ocular disease treatment?

The discovery and development of gene-editing technologies and their applications in managing ocular diseases.

3 Aug 2023

Penn Medicine and CHOP receive $26 million NIH grant to develop treatments for three rare genetic diseases

A Penn Medicine and Children's Hospital of Philadelphia (CHOP) team will seek to develop treatments for three rare, incurable genetic diseases with the help of a $26 million grant from the National Institutes of Health (NIH).

1 Aug 2023

MU researcher receives $3 million grant to use gene editing for investigating the building blocks of disease

In a new study funded by a $3 million grant from the National Institutes of Health (NIH), University of Missouri researcher Kiho Lee, an associate professor in the College of Agriculture, Food and Natural Resources, will use gene editing to investigate the building blocks of disease.

27 Jul 2023

Using CRISPR gene-editing system to build a more sustainable forest

Researchers at North Carolina State University used a CRISPR gene-editing system to breed poplar trees with reduced levels of lignin, the major barrier to sustainable production of wood fibers, while improving their wood properties.

13 Jul 2023

Study provides unprecedented insights into the complexity of large-scale neural networks

That experiences leave their trace in the connectivity of the brain has been known for a while, but a pioneering study by researchers at the German Center for Neurodegenerative Diseases (DZNE) and TUD Dresden University of Technology now shows how massive these effects really are.

11 Jul 2023

As low-nicotine cigarettes hit the market, anti-smoking groups press for wider standard

The idea seems simple enough. Preserve all the rituals of smoking: Light up a cigarette, inhale the smoke, including the nasty stuff that can kill you, and exhale.

30 Jun 2023

First programmable RNA-guided system found in eukaryotes

A team of researchers led by Feng Zhang at the Broad Institute of MIT and Harvard and the McGovern Institute for Brain Research at MIT has uncovered the first programmable RNA-guided system in eukaryotes -; organisms that include fungi, plants, and animals.

28 Jun 2023