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.
Breakthrough gene therapy restores dystrophin protein in Duchenne muscular dystrophy

Breakthrough gene therapy restores dystrophin protein in Duchenne muscular dystrophy

Proof-of-concept animal studies demonstrate a step forward in the fight against HIV

Proof-of-concept animal studies demonstrate a step forward in the fight against HIV

Broken String Biosciences appoints Steve Becker as Chief Commercial Officer

Broken String Biosciences appoints Steve Becker as Chief Commercial Officer

Modulating T-cell response: Innovations in AAV8 gene therapy for retinal diseases

Modulating T-cell response: Innovations in AAV8 gene therapy for retinal diseases

Breakthrough gene therapy offers hope for Duchenne muscular dystrophy

Breakthrough gene therapy offers hope for Duchenne muscular dystrophy

Mogrify appoints Dr. Jonathan Appleby as Chief Scientific Officer

Mogrify appoints Dr. Jonathan Appleby as Chief Scientific Officer

Gene editing silences mutant microRNA, restores hearing in adult mice

Gene editing silences mutant microRNA, restores hearing in adult mice

How lipoproteins shape your metabolic health

How lipoproteins shape your metabolic health

New gene editing technique offers hope for millions

New gene editing technique offers hope for millions

60 years in the making: Nanoparticles revolutionize nucleotide delivery

60 years in the making: Nanoparticles revolutionize nucleotide delivery

UC San Diego Health offers new bladder-saving gene therapy to treat localized bladder cancer

UC San Diego Health offers new bladder-saving gene therapy to treat localized bladder cancer

Small molecule discovery offers hope for treatment of Angelman syndrome

Small molecule discovery offers hope for treatment of Angelman syndrome

TRPC5 gene identified as a diagnostic marker of obesity and postpartum depression

TRPC5 gene identified as a diagnostic marker of obesity and postpartum depression

Cellular Origins acquires Actia Platform IP to enhance automated cell therapy manufacturing

Cellular Origins acquires Actia Platform IP to enhance automated cell therapy manufacturing

Gene therapy milestone at SickKids offers hope for children with SPG50 neurodegenerative disorder

Gene therapy milestone at SickKids offers hope for children with SPG50 neurodegenerative disorder

CHARMs: A new hope for prion disease therapy

CHARMs: A new hope for prion disease therapy

Targeted gene therapy may reduce AML burden and protect the heart from cardiotoxicity

Targeted gene therapy may reduce AML burden and protect the heart from cardiotoxicity

Researchers identify key players in abnormal bone differentiation

Researchers identify key players in abnormal bone differentiation

Comparative study on preventative SMA therapies shows mixed outcomes

Comparative study on preventative SMA therapies shows mixed outcomes

New curative treatment for sickle cell disease shows promising results in international trial

New curative treatment for sickle cell disease shows promising results in international trial

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