POSTS TAGGED AS gene therapy

An Adeno-Associated Virus with Enhanced Ability to Cross the Brain-Blood Barrier

New gene delivery vehicle shows promise for human brain gene therapy
“In a new study published in Science, the team showed [MORE]

Anellovirus Vectors in Gene Therapy

Ring’s ‘new class of viral vector’ is re-dosable in primates, overcoming key challenge of AAVs
“AAVs are the most commonly used [MORE]

Walgreen’s Special Pharmacy Offers Gene Therapy

Walgreens dives into gene and cell therapy delivery
“In a bold move to strategically grow its specialty pharmacy services, Walgreens will [MORE]

Creating a Living Liver Model

“In this work, we [at University of Sydney] describe the application of whole human liver explants in an ex situ [MORE]

Silencing the Bad Cholesterol Gene

Durable and efficient gene silencing in vivo by hit-and-run epigenome editing
“[We, at San Raffaele Telethon Institute for Gene Therapy, Milan, [MORE]

Avian Retrotransposon for Inserting Corrective Genes into the Human Genome

Avian retrotransposon for inserting corrective genes into the human genome.
“Here, we [at UC Berkeley] describe precise RNA-mediated insertion of transgenes [MORE]

Successful Gene Therapy for Congenital Deafness

A medical first- Gene therapy allows boy to hear
“An 11-year-old boy recently became the first person in the U.S. to [MORE]

An RNA Switch for Modulating Gene Expression

An RNA switch for modulating gene expression
“The RNA-based switch controls mammalian gene expression through modulation of a synthetic polyA signal [MORE]

Lipid Nanoparticle Delivery of Anti-Inflammation siRNAs

Lipid nanoparticle delivery of anti-inflammation siRNAs
“Here, [from MIT researchers] several lipid nanoparticle (LNP) formulations are examined, and a lead candidate [MORE]

Gene Therapy in the Brain

Gene therapy can repair neural connections in the brain
“[University of Minnesota] researchers evaluated a new form of gene therapy invented [MORE]

MicroRNA-22 Gene Therapy for Liver Cancer

miR-22 gene therapy treats HCC by promoting anti-tumor immunity and enhancing metabolism
“Our [researchers at the University of California Davis] data [MORE]

One Shot Gene Therapy for Sickle Cell Disease

One Shot Gene Therapy for Sickle Cell Disease
“On July 27 in Science, scientists from Children’s Hospital of Philadelphia and the [MORE]

How Patient Age, Vector Dose, and Immune Response Figure in Gene Therapy for Duchenne Muscular Dystrophy

Gene editing therapy for DMD
“Based on the finding, the researchers [Sarepta Therapeutics] identified a need for more data on the [MORE]

Gene Therapy for Glaucoma

Glaucoma
“The major risk factor for glaucoma is elevated intraocular pressure, which is regulated by the balance between the secretion of [MORE]