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.
UNC gene therapy scientists uncover viral barcode to penetrate blood-brain barrier

UNC gene therapy scientists uncover viral barcode to penetrate blood-brain barrier

Human iPS cell-derived inner ear cells may improve hereditary hearing loss

Human iPS cell-derived inner ear cells may improve hereditary hearing loss

Novel method can edit genes with substantially fewer errors

Novel method can edit genes with substantially fewer errors

Researchers develop gene therapy for idiopathic pulmonary fibrosis

Researchers develop gene therapy for idiopathic pulmonary fibrosis

Stealth gene shuttles open up new avenues to treat aggressive cancers

Stealth gene shuttles open up new avenues to treat aggressive cancers

Researchers use gene therapy that lengthens telomeres to cure pulmonary fibrosis in mice

Researchers use gene therapy that lengthens telomeres to cure pulmonary fibrosis in mice

Researchers describe severe toxicity with AAV9-based high-dose gene therapy in animal models

Researchers describe severe toxicity with AAV9-based high-dose gene therapy in animal models

Chinese researchers develop multifunctional vehicle to transport gene therapeutics

Chinese researchers develop multifunctional vehicle to transport gene therapeutics

Research shows how Zika virus damages placenta to cause malformations in babies

Research shows how Zika virus damages placenta to cause malformations in babies

New vaccine approach offers effective protection against tuberculosis

New vaccine approach offers effective protection against tuberculosis

Researchers discover why exercise is good and high blood pressure is bad for the heart

Researchers discover why exercise is good and high blood pressure is bad for the heart

Sangamo and Pfizer partner for development of new zinc finger protein gene therapy to treat ALS

Sangamo and Pfizer partner for development of new zinc finger protein gene therapy to treat ALS

UC Davis expert expresses caution on genetically engineered HSCs

UC Davis expert expresses caution on genetically engineered HSCs

Genetic labeling helps researchers track behavior of blood cells

Genetic labeling helps researchers track behavior of blood cells

Study suggests potential means of stalling or even reversing diabetes-related blindness

Study suggests potential means of stalling or even reversing diabetes-related blindness

Gene therapy with CAR T-cells could create long-term immunity from HIV

Gene therapy with CAR T-cells could create long-term immunity from HIV

FDA approves new gene therapy targeting rare form of inherited vision loss

FDA approves new gene therapy targeting rare form of inherited vision loss

A guide to Severe Combined Immunodeficiency Disease, review released

A guide to Severe Combined Immunodeficiency Disease, review released

New review provides guidance to doctors who may treat infants with ‘bubble boy disease’

New review provides guidance to doctors who may treat infants with ‘bubble boy disease’

Gene that could be associated with reduced pain response found

Gene that could be associated with reduced pain response found