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.
Novel platform may lead to more improved gene therapies

Novel platform may lead to more improved gene therapies

Gene therapy may be a safe and effective single-dose treatment for hypophosphatasia

Gene therapy may be a safe and effective single-dose treatment for hypophosphatasia

New software tool detects, evaluates, and quantifies off-target editing activity

New software tool detects, evaluates, and quantifies off-target editing activity

New study assesses total economic impact of human genomics

New study assesses total economic impact of human genomics

Lack of functional GAS2 gene causes severe hearing impairment in mice

Lack of functional GAS2 gene causes severe hearing impairment in mice

New gene therapy could help treat Duchenne muscular dystrophy

New gene therapy could help treat Duchenne muscular dystrophy

National DNA Day 2021: An Interview with Professor George Church

National DNA Day 2021: An Interview with Professor George Church

Gene therapy protects eye cells in mice with retinitis pigmentosa

Gene therapy protects eye cells in mice with retinitis pigmentosa

Study shows beneficial effects of gene therapy can be observed even after several decades

Study shows beneficial effects of gene therapy can be observed even after several decades

Study shows how human viruses are better adapted to infect certain tissues

Study shows how human viruses are better adapted to infect certain tissues

First-in-human clinical trial to test safety, efficacy of gene therapy for Alzheimer's disease

First-in-human clinical trial to test safety, efficacy of gene therapy for Alzheimer's disease

New understanding of tumor microenvironment could help enhance immunotherapy

New understanding of tumor microenvironment could help enhance immunotherapy

New AI-based method produces new diversity of AAV capsids for optimized gene therapy

New AI-based method produces new diversity of AAV capsids for optimized gene therapy

Severe genetic immunodeficiency reversed by a rare DNA change

Severe genetic immunodeficiency reversed by a rare DNA change

Gene therapy helps paralyzed mice to walk again

Gene therapy helps paralyzed mice to walk again

Innovative gene therapy approach for regeneration of functional new neurons

Innovative gene therapy approach for regeneration of functional new neurons

New gene therapy approach can improve uterine blood flow in placental insufficiency

New gene therapy approach can improve uterine blood flow in placental insufficiency

Gene therapy and medication can provide certain beneficial effects of physical exercise

Gene therapy and medication can provide certain beneficial effects of physical exercise

A Gene Mutation That Protects Against Disease

A Gene Mutation That Protects Against Disease

New gene therapy can protect visual function in patients with dominant optic atrophy

New gene therapy can protect visual function in patients with dominant optic atrophy