Gene Editing News and Research

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Gene therapy and glycoside drugs offer new hope for polycystic kidney disease treatment

Researchers have shown that dangerous cysts, which form over time in polycystic kidney disease (PKD), can be prevented by a single normal copy of a defective gene.

4 Apr 2024

Gene defect associated with argininosuccinic aciduria corrected using CRISPR-Cas9 technique

Argininosuccinate lyase deficiency (ASLD), also known as argininosuccinic aciduria, is a disease that has been enriched in the Finnish genetic heritage.

4 Apr 2024

Researchers shed light on proteins controlling the development of ovaries in mice

Researchers at the Francis Crick Institute have shed light on the proteins controlling the development of ovaries in mice before and after birth.

22 Mar 2024

Boosting mRNA activity and longevity with multi-tailed structures

Messenger RNA (mRNA) made its big leap into the public limelight during the pandemic, thanks to its cornerstone role in several COVID-19 vaccines

22 Mar 2024

Scientists shed light on key immune regulators involved in Alzheimer’s and other disorders of lipid-rich tissues

Using novel genetic and genomic tools, researchers at the Icahn School of Medicine at Mount Sinai have shed light on the role of immune cells called macrophages in lipid-rich tissues like the brain, advancing our understanding of Alzheimer's and other diseases.

21 Mar 2024

Researchers discover link between FBXW7 mutations and EGFR signaling in colorectal cancer

The Organoid group at the Hubrecht Institute have identified a new link between FBXW7 mutations and EGFR signaling activity.

18 Mar 2024

Ricoh and ERS Genomics enter into CRISPR/Cas9 license agreement

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From ERS Genomics 13 Mar 2024

New “GPS nanoparticle” delivers a genetic punch to the protein implicated in tumor spread

A newly developed "GPS nanoparticle" injected intravenously can home in on cancer cells to deliver a genetic punch to the protein implicated in tumor growth and spread, according to researchers from Penn State.

11 Mar 2024

Breakthrough in CRISPR delivery promises safer gene editing

Recent research in PNAS highlights advancements in non-viral, cell-specific delivery methods for CRISPR-Cas systems, emphasizing their potential to enhance research and gene therapy applications by minimizing risks associated with off-target effects and improving targeted delivery.

7 Mar 2024

Scientists develop robust iPSC-derived rejuvenated T lymphocytes for cervical cancer treatment

Cervical cancer is among the most common malignancies affecting women worldwide. In 2020 alone, approximately 600,000 women were diagnosed with this disease, and over 314,000 died from it.

5 Mar 2024

Researchers take important step toward genetic therapy for hereditary conditions

Researchers who work with tiny drug carriers known as lipid nanoparticles have developed a new type of material capable of reaching the lungs and the eyes, an important step toward genetic therapy for hereditary conditions like cystic fibrosis and inherited vision loss.

5 Mar 2024

New scSNV-seq technique unveils genetic drivers of diseases like cancer and Alzheimer's

Scientists have developed a new screening tool to uncover how genetic changes affect gene activity and can lead to diseases such as cancer, autoimmunity, neurodegeneration and cardiovascular disease. This new tool enables the investigation of thousands of DNA mutations identified by genetic studies in one experiment, guiding the development of advanced diagnostics and treatments.

4 Mar 2024

Breakthrough gene silencing method slashes cholesterol for a year in mice, no DNA damage

Researchers developed an engineered transcriptional repressor that durably silences the Pcsk9 gene in mice, reducing cholesterol levels for up to a year with a single treatment, showcasing a potential breakthrough in genetic disorder treatments without the risks of DNA mutation.

29 Feb 2024

Breakthrough CRISPR technique unlocks insights about cancer immunology

Over the past two decades, the immune system has attracted increasing attention for its role in fighting cancer.

29 Feb 2024

Universal, essential biomarker identified for childhood neuroblastoma

Researchers have newly identified a universal, essential biomarker for the childhood cancer neuroblastoma – and a potential new target for treatment.

27 Feb 2024

Breakthrough gene therapy method utilizes bird retrotransposon for safe gene insertion

The recent approval of a CRISPR-Cas9 therapy for sickle cell disease demonstrates that gene editing tools can do a superb job knocking out genes to cure hereditary disease.

20 Feb 2024

Husker research duo selected as Phase 1 winners in the NIH's Targeted Genome Editor Delivery Challenge

A Husker research duo was named a first-round winner in a National Institutes of Health competition aimed at generating solutions for delivering genome-editing technology to the cells of people with rare and common diseases.

19 Feb 2024

Research demonstrates a bat species' resistance to cancer, pinpoints key genes

A study reveals Myotis pilosus bats exhibit exceptional cancer resistance through gene downregulation, offering insights into natural anticancer mechanisms.

18 Feb 2024

Groundbreaking study sheds light on the potential of the heart to achieve self-repair and regeneration

A groundbreaking scientific study published in Nature Cardiovascular Research has unveiled a remarkable discovery that may have far-reaching implications for the treatment of heart disease.

14 Feb 2024

A century of progress: American Heart Association's historic journey and future directions

Dramatic advances in the understanding and treatment of cardiovascular diseases have saved millions of lives in the 100 years since the founding in 1924 of the American Heart Association, the world's leading voluntary organization focused on heart and brain health for all.

13 Feb 2024