Tag: Gene Therapy

Suppressor tRNAs restored full-length p53 from nonsense-mutant TP53 and, delivered via lipid nanoparticles, cut tumour volume by up to 74 ...

A new review argues that CRISPR antivirals, especially RNA-targeting Cas13 systems, can suppress respiratory viruses in preclinical models, with lung ...

A new review synthesizes the signaling pathways, extracellular matrix cues, metabolic switches and transcription factors that govern cardiomyocyte proliferation and ...

An engineered compact Cas12m variant enables multiplexed CRISPR gene activation from a single AAV vector in mouse models.

A new study finds that the neuronal protein SORT1 preserves endosomal balance, calms microglial inflammation and improves cognition in Alzheimer's ...

A new RNA editing platform called MARRS recruits the cell's own ADAR1 enzyme with engineered circular RNA aptamers, achieving up ...

A qualitative study of 21 adults with familial cardiomyopathy finds that perceived disease severity, family history and safety uncertainties shape ...

A new review details how biomaterials, nanocarriers, gene-activated matrices and smart biosensor dressings are being engineered to restore angiogenesis and ...

A three-year, $1 million National Science Foundation grant will fund Portland State University's eXtreme Virus Lab in its targeted search ...

A systematic review of 58 studies finds that transfusion-based stroke prevention established by the STOP trials remains the cornerstone for ...

A new review maps the molecular, computational and experimental landscape of off-target CRISPR editing as gene therapies advance toward the ...

University of Michigan cardiologists propose a five-question clinical framework for deciding when permanent genomic therapies are justified as gene editing ...

In gerbils, multichannel LED-based optical cochlear implants combined with ChReef gene therapy achieved spectrally distinct auditory midbrain activity at clinically ...

A new narrative review finds that germline mutations underlie up to 20 to 35 percent of pediatric aplastic anemia and ...

A platelet-engineered AAV system that silences the fatty acid transporter CD36 reshaped cardiac lipid metabolism and improved repair after ischemia-reperfusion ...

The 2026 AAO-HNSF meeting will feature research on GLP-1 medications, gene therapy for genetic hearing loss, sleep apnea, cognitive decline, ...

A collaborative publication argues that master protocol trials could make rare neurological disease research more efficient, but it does not ...

A Nature Reviews Bioengineering review examines how tissue engineering, stem cell transplantation and gene-based approaches could advance in utero treatment ...

University of Michigan researchers used long-read nanopore sequencing to resolve hidden cancer-driving alterations in tumors and to trace the origin ...

A single AAV injection carrying the leptin gene normalised fasting insulin and fully reversed hepatic triglyceride accumulation in mice with ...