Gene Editing News and Research

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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

Stem cell gene therapy may offer a promising, curative treatment for sickle cell disease

New research published in the New England Journal of Medicine indicates that stem cell gene therapy may offer a promising, curative treatment for the painful, inherited blood disorder sickle cell disease (SCD).

30 Aug 2023

Drexel University receives grants to support cell and gene therapy education

Drexel University's School of Biomedical Engineering, Science and Health Systems, in collaboration with Drexel's College of Medicine, has received grants from the pharmaceutical company Bristol Myers Squibb, to support the education and training of diverse and talented students looking to pursue careers in cell and gene therapy.

25 Aug 2023

Two small molecule inhibitors can help improve precision, efficiency of CRISPR-Cas9 gene editing

A new study published in Nature Communications demonstrates how small molecule inhibitors can be used to improve the precision and efficiency of CRISPR-Cas9 gene editing.

25 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

Unleashing the Power of Microfluidic Picodroplets

In this interview conducted at SLAS EU 2023 in Brussels, Belgium, we spoke to Richard Hammond, Chief Technical Officer at Sphere Bio, about their microfluidic picodroplet technology and more.

From Fluidic Sciences and Sphere Bio 21 Aug 2023

RARα receptor plays unexpected role in T cell activation

According to new research in the journal Immunity, T cells have a nuclear receptor doing something very odd - but very important - to help them fight pathogens and destroy cancer cells.

18 Aug 2023

Researchers design one-step procedure for improving effective of adoptive cell immunotherapy against solid tumors

Researchers developed a one-step method to engineer multifunctional M1 phenotype macrophages.

14 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

MaxCyte signs strategic platform license with prime medicine to advance next-generation gene editing therapies for patients

MaxCyte, Inc., a leading, cell-engineering focused company providing enabling platform technologies to advance the discovery, development and commercialization of next-generation cell-based therapeutics and to support innovative, cell-based research, today announced the signing of a strategic partnership with Prime Medicine, Inc., a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies.

From MaxCyte, Inc. 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

New gene editing approach could revolutionize the treatment of blood disorders

In a step forward in the development of genetic medicines, researchers at Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

27 Jul 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

Rice bioengineers receive NIH grant for sickle cell disease research

Rice University bioengineer Gang Bao and his team have won a 4-year, $2.6 million grant from the National Institutes of Health to address critical questions surrounding the safety and efficacy of using gene editing to treat sickle cell disease.

18 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

New porcine model could help develop treatments for Dduchenne muscular dystrophy

The group of LMU scientist Eckhard Wolf has developed a porcine DMD model with a mutation, which mimics the hallmarks of the human disease but develops them in an accelerated mode.

12 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