Gene Editing News and Research

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Gene therapy shows dramatic vision improvement for rare inherited condition

The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.

5 Sep 2024

New bacterial defense mechanism revealed as potential tool for genome editing

Scientists who have described in a new study the step-by-step details of a bacterial defense strategy see the mechanism as a promising platform for development of a new genome-editing method.

29 Aug 2024

Hereditary cardiac amyloidosis more common in the UK than previously thought

More people in the UK are at risk of a hereditary form of cardiac amyloidosis, a potentially fatal heart condition, than previously thought, according to a new study led by researchers at UCL (University College London) and Queen Mary University of London.

28 Aug 2024

Exploring the health benefits and production challenges of alternative proteins

Researchers review the health benefits of alternative protein sources and the challenges associated with their production.

20 Aug 2024

New study reveals overlap between metabolic and immune disorders

Inherited diseases of metabolism and immunity have more in common than previously recognized, according to a new study published in the journal Science Immunology.

16 Aug 2024

Columbia study reveals bacteria create free-floating genes, challenging genetic code norms

Since the genetic code was first deciphered in the 1960s, our genes seemed like an open book. By reading and decoding our chromosomes as linear strings of letters, like sentences in a novel, we can identify the genes in our genome and learn why changes in a gene's code affect health.

9 Aug 2024

Using advanced CRISPR to boost efficiency of CAR-T cell therapies

CAR-T cells are highly effective in treating selected blood cancers. However, challenges remain with this new therapy, which was first approved in 2017 in the USA and a year later in Europe for treating acute lymphoblastic leukemia (ALL).

2 Aug 2024

Scientists explore early life factors contributing to SMA

Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, scientists at DZNE and the Dresden University of Technology are now drawing attention to previously unnoticed abnormalities in embryonic development. They base their argument on studies of so-called organoids: Laboratory-grown tissue cultures that can reconstruct disease processes. Their findings are published in the journal Cell Reports Medicine.

27 Jul 2024

Broken String Biosciences appoints Steve Becker as Chief Commercial Officer

Broken String Biosciences (“Broken String”), a genomics company driving development of the next generation of more precise, safe, and effective cell and gene therapies, today announced it has appointed Steve Becker as Chief Commercial Officer.

24 Jul 2024

Common blood thinner can be repurposed as an inexpensive antidote for cobra venom

Scientists at the University of Sydney and Liverpool School of Tropical Medicine have made a remarkable discovery: a commonly used blood thinner, heparin, can be repurposed as an inexpensive antidote for cobra venom.

18 Jul 2024

Harnessing cell's natural delivery system for targeted drug delivery

Each cell in the body has its own unique delivery system that scientists are working on harnessing to move revolutionary biological drugs -; molecules like proteins, RNA and combinations of the two -; to specific diseased parts of the body.

17 Jul 2024

How lipoproteins shape your metabolic health

Study provides a comprehensive review of the lipoprotein pathway and its impact on healthy metabolism, highlighting the roles of HDL, LDL, and other lipoproteins in cardiovascular health and disease.

11 Jul 2024

Prime editing offers promising path to permanent treatment for cystic fibrosis

Cystic fibrosis is one of the most common genetic disorders, causing thick mucus build-up in the lungs and other parts of the body, breathing problems, and infection.

11 Jul 2024

New hope for solid tumors with enhanced CAR-T cells

A promising therapy that treats blood cancers by harnessing the power of the immune system to target and destroy cancer cells could now treat solid tumors more efficiently. Thanks to a recent study published in Molecular Therapy – Methods & Clinical Development from Dan Cappabianca and Krishanu Saha at the Wisconsin Institute for Discovery, Chimeric Antigen Receptor (CAR) T-cell therapy can be improved by altering the conditions the T cells are grown in. And it was all discovered by chance.

3 Jul 2024

Artificial variants of Withanolides act highly specifically against leukemia cells

Nightshade plants produce a diverse array of compounds with therapeutic potential. Researchers at CeMM have now identified an artificial variant inspired by the Withanolides group that acts highly specifically against leukemia cells.

21 Jun 2024

Promising gene editing therapy shows hope in sickle cell disease cure

Researchers have presented the latest findings from a clinical trial aimed at discovering a cure for sickle cell disease, a painful genetic blood disorder with limited treatment options.

14 Jun 2024

Efficient whole-gene insertions can be achieved with improved gene-editing technology

Scientists at the Broad Institute of MIT and Harvard have improved a gene-editing technology that is now capable of inserting or substituting entire genes in the genome in human cells efficiently enough to be potentially useful for therapeutic applications.

10 Jun 2024