Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.
Experimental gene therapy proven safe for preserving vision of people with AMD

Experimental gene therapy proven safe for preserving vision of people with AMD

Spanish scientists develop promising gene therapy method to treat Wilson's disease

Spanish scientists develop promising gene therapy method to treat Wilson's disease

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

Sangamo Therapeutics and Pfizer team up for development of Hemophilia A gene therapy

Sangamo Therapeutics and Pfizer team up for development of Hemophilia A gene therapy

Study: New gene editing strategy effectively targets cancer-causing ‘fusion genes’ in mouse models

Study: New gene editing strategy effectively targets cancer-causing ‘fusion genes’ in mouse models

Latest advances in antibody delivery mediated by rAAV for treatment of chronic and infectious diseases

Latest advances in antibody delivery mediated by rAAV for treatment of chronic and infectious diseases

Researchers use gene-editing tool to restore visual function in mouse models of retinitis pigmentosa

Researchers use gene-editing tool to restore visual function in mouse models of retinitis pigmentosa

Penn researchers pinpoint underlying cause of Best disease

Penn researchers pinpoint underlying cause of Best disease

TSRI scientists find new way to make cell population resistant to HIV

TSRI scientists find new way to make cell population resistant to HIV

Researchers use genetic manipulation to regenerate auditory hair cells in adult mice

Researchers use genetic manipulation to regenerate auditory hair cells in adult mice

Scientists use time-lapse video to identify cells that trigger blood stem cell development

Scientists use time-lapse video to identify cells that trigger blood stem cell development

Researchers use modified AAV as ‘Trojan Horse’ for delivering genes to restore congenital deafness

Researchers use modified AAV as ‘Trojan Horse’ for delivering genes to restore congenital deafness

ISCT honors Dr. Adrian Gee with career achievement award

ISCT honors Dr. Adrian Gee with career achievement award

Researchers achieve new step towards treatment of myotubular myopathy using gene therapy

Researchers achieve new step towards treatment of myotubular myopathy using gene therapy

Thermo Fisher Scientific collaborates with CGT Catapult to create seamless cell and gene therapy supply chain

Thermo Fisher Scientific collaborates with CGT Catapult to create seamless cell and gene therapy supply chain

Inner ear gene therapy holds promise for treating patients with hearing loss and dizziness

Inner ear gene therapy holds promise for treating patients with hearing loss and dizziness

Efficacy of innovative gene therapy can be tested quickly and cost-effectively using new cellular model

Efficacy of innovative gene therapy can be tested quickly and cost-effectively using new cellular model

Innovative strategy uses genetically modified viruses to selectively attack tumor cells

Innovative strategy uses genetically modified viruses to selectively attack tumor cells

Study findings could lead to novel therapies for preventing vision loss from degenerative retinal diseases

Study findings could lead to novel therapies for preventing vision loss from degenerative retinal diseases

Researchers discover new way to curb chronic pain

Researchers discover new way to curb chronic pain