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Zebrafish Model Offers Hope for Treating Ultra-Rare Muscle Disease

Can a small fish help identify possible treatments for an ultra-rare inherited disease found in an Alabama boy? The genetic disease is XMEA, which progressively weakens the muscles and can affect the liver and heart.

6 Jun 2025

Base Editing Breakthrough Brings Hope for Genetic Disease Treatment

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.

5 Jun 2025

PPP2R1A Gene Mutations Improve ATR Inhibitor Drug Effectiveness in Ovarian Clear Cell Cancers

A study in Oncogene shows that PPP2R1A mutations increase sensitivity to ATR inhibitors in ARID1A-mutant ovarian clear cell carcinoma, suggesting a potential biomarker for improved treatment strategies.

5 Jun 2025

Gene Editing Using CRISPR and AAV6 Impacts Long-Term Regeneration of 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.

4 Jun 2025

Repairing DNA Double-Strand Breaks May Improve Genomic Stability by Limiting Organelle-to-Nucleus Gene Transfer

A new study in Nature Plants reveals that DNA double-strand break repair (DSBR) pathways help prevent gene transfer from organelles to the nucleus in plants. Disruption of DSBR increases the risk of genomic instability through endosymbiotic gene transfer (EGT).

2 Jun 2025

Epigenome Editing: Enhancing Endonuclease Protein Design for In Vivo Applications

Researchers have engineered a compact, high-specificity IscB variant, NovaIscB, for safe, persistent in vivo epigenome editing using AAV delivery, offering a powerful tool for gene regulation without DNA breaks.

28 May 2025

New CRISPR Technology for Precise RNA Delivery in Neurons

Proteins produced by RNA segments can aid in the repair of damaged neurons in human bodies.

22 May 2025

Novel Bacterial Immune Weapon that Starves Viral Invaders

The discovery of Cat1 reveals how CARF effectors use complex structures to inhibit viral replication, advancing our understanding of bacterial immune responses.

19 May 2025

Scientists Develop EvoCAST for Efficient and Accurate Gene Insertion

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.

16 May 2025

Depleting TM6SF2 in the Gut Worsens Liver Fat Buildup, Study Finds

A new study finds that gut-specific TM6SF2 depletion exacerbates liver fat accumulation and dysbiosis in mice on high-fat diets, highlighting its importance in gut-liver metabolic regulation.

12 May 2025

Researchers Create DNA Editing Technique to Improve Lactobacillus Strains

A Kobe University team was able to edit the DNA of Lactobacillus strains directly without a template from other organisms.

24 Apr 2025

AI-Powered Protein Engineering for Precise and Scalable Gene Therapy

Genome editing has progressed rapidly, showing promising outcomes for the treatment of genetic disorders, yet there remains potential for enhancement.

23 Apr 2025

Scientists Build Live African Swine Fever Virus From Synthetic DNA Using CRISPR

Utilizing CRISPR-Cas9, scientists reconstitute live African swine fever virus from synthetic genomes, accelerating vaccine development for this deadly virus.

2 Apr 2025

Plants Repurpose Gas Exchange Genes for Improved Defense Against Herbivores

Throughout evolution, plants have continuously adapted to survive in changing environments. Apart from complex structural changes, plants have also developed various defense strategies against herbivores, including tougher protective layers, thorns, and chemical deterrents.

29 Mar 2025

Breakthrough Gene Therapy Shows Promise for Treating Hereditary Hearing Loss

Congenital hearing loss refers to impaired auditory function that occurs due to genetic causes. GJB2 is the gene responsible for approximately half of all cases of hereditary hearing loss.

28 Mar 2025

Scientists Identify Crucial Mechanism for Maintaining Neuronal Identity in the Brain

A team of researchers from the Institute for Neurosciences, a joint center of the Spanish National Research Council (CSIC) and the Miguel Hernández University (UMH) of Elche, in collaboration with researchers from Columbia University (New York, USA), has identified a mechanism that regulates the production of two different proteins from the same gene.

24 Mar 2025