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Personalized gene editing corrects fatal infant metabolic disorder in world-first treatment

Researchers have used a personalised base-editing gene therapy to successfully treat an infant with carbamoyl-phosphate synthetase 1 (CPS1) deficiency, a rare and life-threatening inborn error of metabolism. This pioneering approach chemically corrects the underlying mutation without cutting DNA, showing promise for precision treatment of ultrarare genetic disorders.

20 May 2025

Sapio Sciences introduces Biorepository Management solution in Sapio Release 25.4

Sapio Sciences, the science-aware™ lab informatics platform, today announced Sapio Release 25.4, introducing a new Biorepository Management solution, enhanced sample management capabilities, and platform-wide enhancements designed to streamline lab operations and support high-throughput research environments.

From Sapio Sciences 2 May 2025

Novel cancer immunotherapy approach could complement existing CAR-T treatments

Researchers in South Korea have proposed a novel cancer immunotherapy approach that could complement existing CAR-T (Chimeric Antigen Receptor T-cell) treatments.

24 Apr 2025

Small peptides play big role in halting bacterial translation

Proteins form the structural and functional backbone of the cell, and any perturbation in their synthesis can disrupt normal cellular functions.

18 Apr 2025

Unexpected function of gene network in gut microbes revealed

Could a gene regulatory network in gut microbes have evolved its elaborate and tightly regulated molecular machinery only to pump out antibiotics indiscriminately? Researchers from the Institute of Science and Technology Austria (ISTA) show this is an auxiliary function.

8 Apr 2025

TRIMTECH Therapeutics raises $31M seed funding to advance targeted protein degradation pipeline for treatment of neurodegenerative diseases

TRIMTECH Therapeutics raises $31M to enhance its TRIMTAC platform, targeting protein aggregates in neurodegenerative diseases for improved therapeutic outcomes.

From TRIMTECH Therapeutics 7 Mar 2025

Yale rewrites genetic code for new proteins

Synthetic biologists from Yale were able to re-write the genetic code of an organism - a novel genomically recoded organism (GRO) with one stop codon - using a cellular platform that they developed enabling the production of new classes of synthetic proteins. These synthetic proteins, researchers say, offer the promise of innumerable medical and industrial applications that can benefit society and human health.

11 Feb 2025

Study identifies potential gain of function mutation in retinoblastoma

A new research paper was published in Volume 16 of Genes & Cancer on January 20, 2025, entitled, "Analysis of pathogenic variants in retinoblastoma reveals a potential gain of function mutation."

29 Jan 2025

New genetic syndrome bridges the gap between neurodevelopmental and neurodegenerative disorders

In a landmark discovery, researchers from the University of Antioquia have identified a new genetic syndrome that bridges the gap between neurodevelopmental disorders and neurodegenerative diseases.

30 Sep 2024

Scripps Research develops breakthrough technique for adding unique amino acids to proteins

It's a dogma taught in every introductory biology class: Proteins are composed of combinations of 20 different amino acids, arranged into diverse sequences like words.

11 Sep 2024

RNA Therapeutics Core launches at Mass General Brigham to boost circular RNA research

The Mass General Brigham Gene and Cell Therapy Institute (GCTI) today announced it has launched the RNA Therapeutics Core, a first-of-its-kind, state-of-the-art facility and resource to advance the use of RNA technologies within and beyond the Mass General Brigham research ecosystem.

3 Sep 2024

Intranasal vaccine shows broad SARS-CoV-2 variant protection

Study demonstrates that a single-dose intranasal live-attenuated vaccine, CDO-7N-1, provides broad protection against SARS-CoV-2 and its variants by inducing potent immune responses, including neutralizing antibodies and T-cell activity, in animal models.

1 Sep 2024

New intranasal COVID-19 vaccine promises broad protection and accessibility

A next-generation COVID-19 mucosal vaccine is set to be a gamechanger not only when delivering the vaccine itself, but also for people who are needle-phobic.

27 Aug 2024

Research uncovers dengue’s surprising invasion strategies

Mosquito-borne viral infections once confined to tropical regions are spreading. Dengue virus infects up to 400 million people worldwide each year according to World Health Organization estimates, and no available treatments exist for this disease.

22 Jul 2024

deCODE genetics study highlights potential drug targets for autoimmune thyroid disease

Scientists at deCODE genetics, a subsidiary of Amgen, have published a study in Nature Communications, comparing over 110 thousand patients with autoimmune thyroid disease (AITD) from Iceland, Finland, UK and USA with 1,1 million controls.

11 Jul 2024

Scientists identify over 5,000 genetic variants that enable certain cancers to thrive

Researchers performed saturation genome editing of the BAP1, whose dysfunction is related to cancer and impaired neurodevelopment.

10 Jul 2024

Study shows the benefits of locally administered intranasal live-attenuated SARS-CoV-2 vaccine

Rocketvax AG, together with the German Research Foundation (DFG), has financially supported a study carried out as part of a joint project of the Swiss National Science Foundation (SNSF, National Research Program "Covid-19," NRP 78) and the German Research Foundation (DFG) "Recoding the SARS-CoV-2 Genome - A Multidisciplinary Approach to Generate Live-Attenuated Coronavirus Vaccines."

27 Feb 2024