Gene Editing News and Research

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Blocking a single gene disrupts vitamin D metabolism and cancer pathways in new cell study

Researchers identified SDR42E1 as a key regulator of vitamin D absorption and sterol metabolism in colorectal cancer cells. Disruption of this enzyme altered cancer-related pathways and cell viability, highlighting its potential as a therapeutic target.

21 Jul 2025

Prime editing reverses symptoms of severe childhood neurological disease

Scientists use a precise form of gene editing called prime editing to correct the most common genetic mutations that cause alternating hemiplegia of childhood, a rare and severe neurological disorder that begins in infancy.

21 Jul 2025

Gene editing in the brain offers hope for ultra-rare neurological disease

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a single injection, a feat with profound implications for patients with neurological diseases.

21 Jul 2025

Zebrafish reveal how two genes independently drive sensory organ regeneration

Researchers identified that two cyclin D genes, ccndx and ccnd2a, independently regulate distinct stem and progenitor cell populations during zebrafish sensory organ regeneration. The findings reveal that regeneration can proceed via alternative pathways even when one gene is disrupted, providing new insight into tissue repair mechanisms.

20 Jul 2025

Developing AI-driven stem cell therapies to fight ovarian aging

Ovarian aging, characterized by diminishing egg reserves and quality, affects millions of women worldwide, often leading to infertility.

18 Jul 2025

Old chemistry unlocks safer and stronger mRNA delivery

As millions of people know firsthand, the most common side effect of mRNA vaccines like the COVID-19 shot is inflammation: soreness, redness and a day or two of malaise.

18 Jul 2025

Study identifies SDR42E1's role in vitamin D pathway

Vitamin D is not only an essential nutrient, but also the precursor of the hormone calcitriol, indispensable for health: it regulates the uptake of phosphate and calcium necessary for bones by the intestines, as well as cell growth and the proper function of muscles, nerve cells, and the immune system.

18 Jul 2025

New funding brings personalized CRISPR cures closer to children with rare diseases

Personalized CRISPR cures for children born with rare genetic diseases are now a step closer to being more widely available.

8 Jul 2025

Landmark discovery reshapes understanding of key cancer protein MCL-1

Landmark research on MCL-1, a critical protein that is an attractive target for cancer drug development, helps explain why some promising cancer treatments are causing serious side effects, and offers a roadmap for designing safer, more targeted therapies.

8 Jul 2025

New gene editing approach targets mutation behind common genetic heart disease

Fourteen million people worldwide suffer from enlarged hearts, or hypertrophic cardiomyopathy (HCM), a genetic disease that thickens the heart's walls, making it harder for the organ to pump blood - but many of them don't know it.

8 Jul 2025

Brown University scientists discover protein essential for heart survival

New findings by a team of molecular biologists at Brown University on the critical role of a protein called AIMP3 in heart function could inform new treatments for heart disease.

25 Jun 2025

UNIGE scientists uncover two key proteins that regulate chromatin remodelling

Cells depend on the precise reading of DNA sequences to function correctly. This process, known as gene expression, determines which genetic instructions are activated.

24 Jun 2025

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