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.
Nanoparticles mimicking flu viruses to deliver large biological drug molecules

Nanoparticles mimicking flu viruses to deliver large biological drug molecules

CRISPR introduces potentially dangerous changes in cells, reveals research

CRISPR introduces potentially dangerous changes in cells, reveals research

Researchers employ hepatic organoids for disease modeling

Researchers employ hepatic organoids for disease modeling

MIP Diagnostics to support the development of challenging affinity reagents with new Molecular Imprinting Epitope Discovery

MIP Diagnostics to support the development of challenging affinity reagents with new Molecular Imprinting Epitope Discovery

'Switching on' Sight using Gene Therapy

'Switching on' Sight using Gene Therapy

Study uncovers new gene variants that may cause inherited retinal dystrophies

Study uncovers new gene variants that may cause inherited retinal dystrophies

Pioneering gene therapy has long-lasting effects on children with deadly immune disorder

Pioneering gene therapy has long-lasting effects on children with deadly immune disorder

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