Gene Editing News and Research

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Cedars-Sinai study finds new way to combat Alzheimer’s in preclinical models

In a preclinical study, Cedars-Sinai investigators found that boosting angiotensin converting enzyme (ACE) in key immune cells called microglia in the brain helped protect the brains of specially bred laboratory mice against Alzheimer's disease.

6 Aug 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.

From The Jackson Labs 6 Aug 2025

Apple snail eye regeneration offers new insights into human sight restoration

The eye of the apple snail is unusually similar to a human eye-but, unlike human eyes, it can regrow itself if injured or even amputated.

6 Aug 2025

Targeting lactate metabolism offers new avenues for cancer treatment

Lactic acid (LA) has transitioned from being perceived as a mere glycolytic waste product to a pivotal regulator of tumor-immune crosstalk.

5 Aug 2025

Researchers classify how specific genetic mutations correspond to rare disorders

The NF-κB signaling pathway plays a pivotal role in how our bodies fight infection, heal, and regulate inflammation. At the heart of this pathway is the NEMO protein, encoded by the IKBKG gene.

30 Jul 2025

New RNA therapy reverses symptoms of progeria in mouse models

Children who develop deep wrinkles, stunted growth, and rapidly aging bones and blood vessels as early as 1 to 2 years of age may be suffering from Hutchinson-Gilford Progeria Syndrome (HGPS), a rare and incurable genetic disorder that affects approximately one in eight million people.

29 Jul 2025

New method genetically blocks mosquitoes from transmitting malaria

Mosquitoes kill more people each year than any other animal. In 2023, the blood-sucking insects infected a reported 263 million people with malaria, leading to nearly 600,000 deaths, 80% of which were children.

23 Jul 2025

Astrocytes play key role in coordinating neurons for visual information

Cells called astrocytes are about as abundant in the brain as neurons, but scientists have spent much less time figuring out how they contribute to brain functions.

22 Jul 2025

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

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