Tag: Gene Therapy

A University of Cincinnati and UC Davis team showed that an AAV gene therapy targeting cerebrospinal fluid-producing cells reduced hydrocephalus-related ...

MIT researchers have engineered ligand-directed lipid nanoparticles that reprogram natural killer cells into CAR-bearing cancer killers directly inside living animals, ...

A new Pediatric Research reflection by Alex Smith traces nearly twenty years of living alongside his son Harrison's Duchenne muscular ...

A new review charts how stem cell transplantation, lentiviral gene therapy, and CRISPR-based editing such as Casgevy are turning beta-thalassemia ...

An international trial led by University of Pittsburgh scientists found that an optogenetic gene therapy combined with light-projecting goggles was ...

New research shows that the telomerase enzyme TERT drives neural repair after stroke and that the herbal compound cycloastragenol can ...

Researchers converted Duchenne patient stem-cell teratomas into humanized models in which both gene-corrected cell transplants and lipid nanoparticle base editing ...

A new review confirms that beremagene geperpavec, the first FDA-approved topical gene therapy, significantly improves wound healing in dystrophic epidermolysis ...

A new review in Virology Journal examines how siRNA and CRISPR/Cas gene-silencing technologies could target the HPV16 oncoproteins E5 and ...

University of Zurich researchers found that a one-hour treatment with activated protein C keeps human blood stem cells in a ...

A new Pediatric Research commentary argues that insurance coverage and reimbursement policy, not the underlying science, now determine which children ...

A five-year clinical cohort study shows that an optogenetic therapy combining gene injection and light-stimulating goggles is safe and enabled ...

A new review argues that Huntington's disease is driven by widespread disruptions of interconnected protein networks, pointing to hub proteins ...

A new review charts the rapid rise of AAV-mediated OTOF gene replacement therapy, from the first human trials to the ...

In a groundbreaking study published in Gene Therapy, researchers have illuminated the possibilities of gene editing for collagen disorders, highlighting ...

In a groundbreaking study, researchers have unveiled a potential breakthrough in the treatment of Amyotrophic Lateral Sclerosis (ALS) through innovative ...

In the realm of gene therapy, the assessment of neutralizing antibodies against adeno-associated viruses (AAVs) has become a pivotal focal ...

In recent years, the scientific community has made significant strides in the understanding and treatment of collagen disorders, a diverse ...

In the quest to develop effective therapies for cardiac diseases, researchers are increasingly turning their attention to lipid nanoparticle (LNP)−mRNA ...

For many years, cardiomyopathy has been shrouded in complexity, representing a constellation of heart muscle disorders that significantly impact millions ...