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.
Thermo Fisher Scientific collaborates with CGT Catapult to create seamless cell and gene therapy supply chain

Thermo Fisher Scientific collaborates with CGT Catapult to create seamless cell and gene therapy supply chain

Inner ear gene therapy holds promise for treating patients with hearing loss and dizziness

Inner ear gene therapy holds promise for treating patients with hearing loss and dizziness

Efficacy of innovative gene therapy can be tested quickly and cost-effectively using new cellular model

Efficacy of innovative gene therapy can be tested quickly and cost-effectively using new cellular model

Innovative strategy uses genetically modified viruses to selectively attack tumor cells

Innovative strategy uses genetically modified viruses to selectively attack tumor cells

Study findings could lead to novel therapies for preventing vision loss from degenerative retinal diseases

Study findings could lead to novel therapies for preventing vision loss from degenerative retinal diseases

Researchers discover new way to curb chronic pain

Researchers discover new way to curb chronic pain

Study identifies protein that may offer protection against heart disease

Study identifies protein that may offer protection against heart disease

Study describes method to engineer brown adipose tissue from white fat-derived stem cells

Study describes method to engineer brown adipose tissue from white fat-derived stem cells

Innovative gene therapy allows complete remission of clinical signs of sickle cell disease

Innovative gene therapy allows complete remission of clinical signs of sickle cell disease

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