Gene Editing News and Research

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New approach to combat cancer by reactivating natural RNA kill switch

Alternative RNA splicing is like a movie editor cutting and rearranging scenes from the same footage to create different versions of a film.

14 Mar 2025

CRISPR technologies paving the way for advances in regenerative medicine

A recent review published in the journal Engineering delves into the significant advancements and potential of CRISPR technologies in the field of regenerative medicine.

13 Mar 2025

Study uncovers how transposons use telomeres for survival in bacteria

Transposons, or "jumping genes" – DNA segments that can move from one part of the genome to another – are key to bacterial evolution and the development of antibiotic resistance.

7 Mar 2025

New CRISPR-enriched metagenomics method for detecting antibiotic resistance genes in wastewater

Antibiotic resistance is a global concern that threatens our ability to prevent and treat bacterial infections in humans and animals.

4 Mar 2025

Are Allogeneic CAR-Ts the Future of LBCL Cancer Treatment? - Making Safe, Durable & Effective Universal Cell Therapies a Reality

Allogene Therapeutics recently released exciting results from the Phase 1 ALPHA/ALPHA2 trials of the Allogeneic CAR T 'Cemacabtagene Ansegedleucel/ALLO-501' in Relapsed/Refractory Large B-Cell Lymphoma.

From Hanson Wade Group - 7th Allogeneic Cell Therapies Summit 4 Mar 2025

New CRISPR-based test enhances diagnosis of life-threatening fungal infection

Tulane University researchers have developed a CRISPR-based test that diagnoses Pneumocystis jirovecii pneumonia (PJP) - a life-threatening fungal infection primarily affecting children and immunocompromised patients - more quickly and less invasively, according to a new study published in the Journal of Clinical Investigation.

3 Mar 2025

Pioneering the world’s first CRISPR medicine for sickle cell disease

When Vijay Sankaran was an MD-PhD student at Harvard Medical School in the mid-2000s, one of his first clinical encounters was with a 24-year-old patient whose sickle cell disease left them with almost weekly pain episodes.

3 Mar 2025

St. Jude scientists discover how retinoic acid selectively kills metastatic neuroblastoma cells

Neuroblastoma is a solid tumor that occurs in children. When high-risk, the disease has a poor prognosis. Decades ago, adding the drug retinoic acid to neuroblastoma treatment increased survival by 10-15%.

2 Mar 2025

Study demonstrates successful gene therapy for maple syrup urine disease

A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored coordinated expression of the affected genes and stabilized biomarkers in a calf as well as in mice.

28 Feb 2025

Researchers make breakthrough in understanding the role of tau protein in Alzheimer's

A research team at the University of Cologne has made a significant breakthrough in understanding the role of the tau protein in Alzheimer's disease.

28 Feb 2025

Cambridge launches program to tackle engineered pandemic risks

Covid-19 showed us how vulnerable the world is to pandemics – but what if the next pandemic were somehow engineered? How would the world respond – and could we stop it happening in the first place?

27 Feb 2025

Genetic mutation offers hope for better rheumatoid arthritis treatments

Research led by York University's Faculty of Health reveals how a specific mutation in a protein called TRAF1 can shut down an overactive immune response, dramatically reducing inflammation in mice.

27 Feb 2025

Gene editing shows promise for treating trisomy at cellular level

Gene editing techniques may eventually allow trisomy to be treated at the cellular level, according to an in vitro proof-of-concept study.

18 Feb 2025

Human-specific gene variant may hold key to evolution of language

The origins of human language remain mysterious. Are we the only animals truly capable of complex speech? Are Homo sapiens the only hominids who could give detailed directions to a far-off freshwater source or describe the nuanced purples and reds of a dramatic sunset?

18 Feb 2025

Stanford researchers identify key variants linked to inherited cancer risk

Thousands of single changes in the nucleotides that make up the human genome have been associated with an increased risk of developing cancer.

17 Feb 2025

Discovery of gene silencer offers hope for autosomal dominant leukodystrophy

A team led by University of Pittsburgh School of Public Health geneticists has shown, for the first time, that a gene "silencer" that resides in junk DNA is directly sparing people from a devastating and fatal progressive neurological disease.

13 Feb 2025

One shot to lower cholesterol for life? Scientists unlock a groundbreaking gene therapy

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction without altering DNA, offering a potential one-time treatment for high cholesterol.

11 Feb 2025

MaxCyte® acquires SeQure Dx to broaden cell engineering offerings with on-target and off-target editing assessments

MaxCyte, Inc., a leading, cell-engineering focused company providing solutions to advance the discovery, development and commercialization of next-generation cell therapeutics announced today the acquisition of SeQure Dx, a market leader of on-target and off-target editing assessment services for cell and gene therapies.

From MaxCyte, Inc. 6 Feb 2025

Breakthrough drug improves treatment of medulloblastoma in mice

Brain cancer is the second-leading cause of death in children in the developed world. For the children who survive, standard treatments have long-term impacts on their development and quality of life, particularly in small children and infants.

4 Feb 2025

Engineered fat cells starve tumors and prevent cancer growth

Scientists transformed energy-storing white fat cells into calorie-burning 'beige' fat. Once implanted, they outcompeted tumors for resources, beating back five different types of cancer in lab experiments.

4 Feb 2025