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.
Cedars-Sinai researchers move closer to developing biological pacemaker with new $3 million NIH grant

Cedars-Sinai researchers move closer to developing biological pacemaker with new $3 million NIH grant

Scientists engineer smallest CRISPR-Cas9 system to date

Scientists engineer smallest CRISPR-Cas9 system to date

Glowing mice could mark major step forward for new gene therapy

Glowing mice could mark major step forward for new gene therapy

Canadian researchers use gene therapy to treat patient with Fabry disease

Canadian researchers use gene therapy to treat patient with Fabry disease

New report highlights serious health problems faced by spinal cord injury patients

New report highlights serious health problems faced by spinal cord injury patients

Gene therapy treats myotubular myopathy in dogs

Gene therapy treats myotubular myopathy in dogs

Signals from fat cells may aid in diagnostics for metabolic conditions

Signals from fat cells may aid in diagnostics for metabolic conditions

Penn Medicine's ODC collaborates with FAST to develop gene therapy for Angelman syndrome

Penn Medicine's ODC collaborates with FAST to develop gene therapy for Angelman syndrome

Scientists identify how imbalance of calcium in mitochondria may contribute to Alzheimer's disease

Scientists identify how imbalance of calcium in mitochondria may contribute to Alzheimer's disease

Review shows lipid nanoparticles as highly promising delivery systems in gene therapy

Review shows lipid nanoparticles as highly promising delivery systems in gene therapy

Northwestern Medicine scientists discover retinal cell that may cause myopia

Northwestern Medicine scientists discover retinal cell that may cause myopia

Research unravels molecular mechanisms of antisense oligonucleotides

Research unravels molecular mechanisms of antisense oligonucleotides

Study reveals new molecular mechanism vital for associative memory encoding in the hippocampus

Study reveals new molecular mechanism vital for associative memory encoding in the hippocampus

Scientists restore partial hearing and balance in deaf mice using new gene therapy

Scientists restore partial hearing and balance in deaf mice using new gene therapy

New MIP system for therapeutic gene delivery can enhance transgene expression using AAV vector

New MIP system for therapeutic gene delivery can enhance transgene expression using AAV vector

New gene therapy for Pompe disease may replace currently available treatments

New gene therapy for Pompe disease may replace currently available treatments

Novel gene therapy restores partial hearing and balance in mice born with genetic deafness

Novel gene therapy restores partial hearing and balance in mice born with genetic deafness

Salk Institute researchers reveal how to curb immune enthusiasm

Salk Institute researchers reveal how to curb immune enthusiasm

MedCision announce participation at upcoming conference on cell and gene therapy

MedCision announce participation at upcoming conference on cell and gene therapy

Orphan Disease Center establishes new initiative that focuses on ALS

Orphan Disease Center establishes new initiative that focuses on ALS