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.
Researchers identify the mechanism leading to deafness in Norrie disease

Researchers identify the mechanism leading to deafness in Norrie disease

Gene therapy turns glial brain cells into neurons, restores visual function

Gene therapy turns glial brain cells into neurons, restores visual function

Supercoiling and looping can transmit mechanical stress along the DNA backbone, shows study

Supercoiling and looping can transmit mechanical stress along the DNA backbone, shows study

Exploring cell and gene based therapies for Parkinson’s disease

Exploring cell and gene based therapies for Parkinson’s disease

Researchers develop novel cell reprogramming strategy to slow down glioblastomas

Researchers develop novel cell reprogramming strategy to slow down glioblastomas

Study explores the efficiency of CasMINI, an engineered CRISPR-Cas system

Study explores the efficiency of CasMINI, an engineered CRISPR-Cas system

Miniature CRISPR-Cas system expands applicability and implications further

Miniature CRISPR-Cas system expands applicability and implications further

New study could help develop novel treatments for neurodegenerative diseases

New study could help develop novel treatments for neurodegenerative diseases

Modification in some RNA molecules could protect against non-alcoholic fatty liver

Modification in some RNA molecules could protect against non-alcoholic fatty liver

Gene therapy may be safe and effective in treating AADC deficiency

Gene therapy may be safe and effective in treating AADC deficiency

Novel gene therapy offers hope to children with genetic, neurodegenerative diseases

Novel gene therapy offers hope to children with genetic, neurodegenerative diseases

Children with rare genetic disorder experience dramatic improvements in a gene therapy trial

Children with rare genetic disorder experience dramatic improvements in a gene therapy trial

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