Gene Editing News and Research

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New KRAS mutation discovery opens door to targeted colon cancer therapies

Scientists at Fox Chase Cancer Center have identified a mutation in a common oncogene - a gene that can cause cancer - that may play an important role in colon cancer. Researchers also showed how this mutation could be targeted therapeutically.

24 Jun 2025

Targeting ST6Gal-I shows promise in reducing Alzheimer’s disease pathology

A recent study published in Engineering has provided novel insights into the involvement of α2,6-sialylation in Alzheimer's disease (AD), revealing that the ablation of α2,6-sialyltransferase-I (ST6Gal-I), an enzyme responsible for α2,6-sialylation, can downregulate BACE1 expression and suppress the production of amyloid-β42 (Aβ42) plaques, which are hallmarks of AD.

20 Jun 2025

Fibroblast Growth Factor 8 drives key processes in embryonic development

The role of Fibroblast Growth Factor 8 (FGF8) in embryonic development and organ formation is gaining increased recognition as scientists uncover its wide-ranging influence.

20 Jun 2025

Circular RNAs emerge as key players in hepatitis B-related liver cancer

Hepatitis B virus-induced hepatocellular carcinoma (HCC) remains a major global health burden, and new therapeutic and diagnostic approaches are urgently needed.

20 Jun 2025

NUCDF launches national project to advance urea cycle disorder research

The National Urea Cycle Disorders Foundation, the leading patient advocacy group for people affected by urea cycle disorders (UCDs), today announced the launch of a two-year national project to establish the multistakeholder NUCDF Partner Network and develop a roadmap for future research in these rare disorders.

19 Jun 2025

Yale researchers triple precision and efficiency in genome editing

The promise of genome editing to help understand human diseases and create new therapies is vast, but technological limitations have limited advancement of the field.

18 Jun 2025

Autophagy boosts precision in gene editing

Precision gene editing is crucial for treating genetic diseases, as it enables targeted correction of specific mutations.

12 Jun 2025

Patient-derived xenograft models may become an indispensable tool in precision oncology

Cancer remains one of the most pressing global health challenges, with millions of new diagnoses each year. While precision medicine and targeted therapies have transformed treatment strategies, many patients still face drug resistance and disease recurrence.

10 Jun 2025

Study offers new insights into how the body clears dead cells during stress

A new study from The University of Texas at Arlington details a novel strategy for how the body clears out dead cells during stress, revealing unexpected roles for well-known stress-response genes-a discovery that could help scientists better understand diseases affecting the immune system, brain and metabolism.

9 Jun 2025

Breakthrough drug delivery system transports genetic therapies directly to the lungs

Scientists have made a key breakthrough for treating respiratory diseases by developing a new drug delivery system that transports genetic therapies directly to the lungs, opening promising possibilities for patients with conditions like lung cancer and cystic fibrosis.

5 Jun 2025

Gene editing can trigger inflammatory, senescence-like responses in blood stem cells

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using CRISPR-Cas9 in combination with AAV6 vectors can trigger inflammatory and senescence-like responses in blood stem cells, compromising their long-term ability to regenerate the blood system.

5 Jun 2025

Understanding how base editing tools work at the molecular level

You may have seen it in the news recently: a baby in Pennsylvania with a rare genetic disorder was healed with a personalized treatment that repaired his specific genetic mutation.

4 Jun 2025

US funding freeze halts malaria prevention and genomic research

The “catastrophic” freeze on US funding for malaria has halted prevention programmes across Africa and also threatens to stall advances in genomic research, says Jane Carlton, director of the Johns Hopkins Malaria Research Institute.

1 Jun 2025

Universal CAR T cell therapy shows promise for aggressive T cell cancers

A new type of immunotherapy that targets aggressive blood cancers shows promising results alongside manageable side effects, according to the results of an international phase 1/2 clinical trial led by researchers at Washington University School of Medicine in St. Louis.

31 May 2025

Early allergen introduction reduces childhood food allergy risk

Investigating allergy prevention, this research highlights early intervention strategies and the promise of biologic treatments in managing allergic conditions.

29 May 2025

NUS researchers develop breakthrough gene delivery technology for immune cells

Researchers at the National University of Singapore (NUS) have developed a scalable, non-viral technology that efficiently delivers genetic material into human immune cells.

26 May 2025

New CRISPR technology delivers RNA precisely to repair damaged brain cells

When a neuron in our body gets damaged, segments of RNA produce proteins that can help repair the injury.

21 May 2025

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

Genetic variations in Prader-Willi syndrome offer key insights into autism and psychosis

In a comprehensive Genomic Press Invited Expert Review, researchers from the University of Haifa have synthesized cutting-edge findings on Prader-Willi syndrome (PWS), revealing how this complex neurodevelopmental disorder serves as a critical model for understanding the interplay between autism spectrum disorder (ASD) and psychotic spectrum disorders (PSD).

20 May 2025

New gene editor evoCAST enables precise insertion of complete genes

Ask scientists what gene editing tool is most needed to advance gene therapy, and they'd probably describe a system that's now close to realization in the labs of Samuel Sternberg at Columbia University Vagelos College of Physicians and Surgeons and David Liu at the Broad Institute of MIT and Harvard.

15 May 2025