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.
Gene therapy protects optic nerve cells and preserves vision in glaucoma mouse models

Gene therapy protects optic nerve cells and preserves vision in glaucoma mouse models

Investigational Oncomagnetic Device shrinks end-stage recurrent glioblastoma

Investigational Oncomagnetic Device shrinks end-stage recurrent glioblastoma

Chemical modification in RNA molecules may offer protection against liver disease

Chemical modification in RNA molecules may offer protection against liver disease

Gene therapy delivered into the brain may be safe, effective in treating rare neurological disorder

Gene therapy delivered into the brain may be safe, effective in treating rare neurological disorder

Researchers study the role of subnuclear NSrp70 in T cell-mediated adaptive immunity

Researchers study the role of subnuclear NSrp70 in T cell-mediated adaptive immunity

Yale School of Public Health and Tevogen Bio partner to expand school's regulatory sciences program

Yale School of Public Health and Tevogen Bio partner to expand school's regulatory sciences program

Curia to Acquire US-based LakePharma, Expanding End-to-End R&D Capabilities and Scale for Biologics

Curia to Acquire US-based LakePharma, Expanding End-to-End R&D Capabilities and Scale for Biologics

Gene therapy shows promise in children with devastating genetic disorder

Gene therapy shows promise in children with devastating genetic disorder

Novel method of gene therapy promises to transform the treatment of rare genetic disorder

Novel method of gene therapy promises to transform the treatment of rare genetic disorder

Study: Gene editing therapy for early-stage Huntington’s disease may have long-lasting benefits

Study: Gene editing therapy for early-stage Huntington’s disease may have long-lasting benefits

Nanotechnology and the fight against COVID-19

Nanotechnology and the fight against COVID-19

Preliminary results of AAV-mediated gene therapy for Crigler Najjar syndrome

Preliminary results of AAV-mediated gene therapy for Crigler Najjar syndrome

RDM Positive Impact Foundation funds $1.25 million research project to study SYNGAP1

RDM Positive Impact Foundation funds $1.25 million research project to study SYNGAP1

Researchers design a smart device that delivers medications to prevent HIV transmission

Researchers design a smart device that delivers medications to prevent HIV transmission

Repressing a protein that facilitates DNA repair to potentiate chemotherapy

Repressing a protein that facilitates DNA repair to potentiate chemotherapy

New approach to gene therapy uses common pain reliever to fight against genetic diseases

New approach to gene therapy uses common pain reliever to fight against genetic diseases

Researchers identify a common mechanism underlying diverse brain disorders

Researchers identify a common mechanism underlying diverse brain disorders

Experimental monoclonal antibody could be a new weapon against HIV epidemic

Experimental monoclonal antibody could be a new weapon against HIV epidemic

Gene therapy may be a safe and effective single-dose treatment for soft bone disease

Gene therapy may be a safe and effective single-dose treatment for soft bone disease

Researchers create cellular and spatial “atlas” of the choroid plexus during different life stages

Researchers create cellular and spatial “atlas” of the choroid plexus during different life stages