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 use gene therapy to restore hearing and balance in mouse model of Usher syndrome

Researchers use gene therapy to restore hearing and balance in mouse model of Usher syndrome

Novel gene therapy can inhibit, reverse multiple sclerosis in mouse models

Novel gene therapy can inhibit, reverse multiple sclerosis in mouse models

Gene therapy could be novel approach to treat alcoholism, research suggests

Gene therapy could be novel approach to treat alcoholism, research suggests

Scientists take important step toward gene therapy for deaf patients

Scientists take important step toward gene therapy for deaf patients

Research could lead to improvements in treating diseases caused by mutations in genes

Research could lead to improvements in treating diseases caused by mutations in genes

MSC transplantation can be safe, effective approach to treat Crohn's disease-related fistula

MSC transplantation can be safe, effective approach to treat Crohn's disease-related fistula

MGH researchers identify potential strategy for improving efficacy of angiogenesis inhibitors

MGH researchers identify potential strategy for improving efficacy of angiogenesis inhibitors

New testing regime could speed up diagnosis and treatment of SCID in infants

New testing regime could speed up diagnosis and treatment of SCID in infants

CGT Catapult partners with TrakCel to incorporate tracking technology into cell and gene therapy manufacturing centre

CGT Catapult partners with TrakCel to incorporate tracking technology into cell and gene therapy manufacturing centre

Penn researchers find abnormally shortened telomeres in young DMD patients’ muscle stem cells

Penn researchers find abnormally shortened telomeres in young DMD patients’ muscle stem cells

UChicago Medicine working with Novartis to offer breakthrough gene therapy for pediatric ALL patients

UChicago Medicine working with Novartis to offer breakthrough gene therapy for pediatric ALL patients

Feeder-free stem cell culture media now available from AMSBIO

Feeder-free stem cell culture media now available from AMSBIO

FDA makes the first gene therapy Kymriah available for treatment of pediatric ALL

FDA makes the first gene therapy Kymriah available for treatment of pediatric ALL

Pioneering cancer gene therapy gets green light — and $475,000 price tag

Pioneering cancer gene therapy gets green light — and $475,000 price tag

FDA approves first cell-based gene therapy for use in the United States

FDA approves first cell-based gene therapy for use in the United States

Immunotherapy that uses existing cancer drugs in a new way delivers promising results

Immunotherapy that uses existing cancer drugs in a new way delivers promising results

Junk genes could one day offer framework for treating people with familial hypercholesterolemia

Junk genes could one day offer framework for treating people with familial hypercholesterolemia

Researchers identify important adenovirus receptor in macrophages of mice

Researchers identify important adenovirus receptor in macrophages of mice

Emory scientists use new tools to detect granulins for the first time inside cells

Emory scientists use new tools to detect granulins for the first time inside cells

New version of gene editing technique corrects molecular mistakes in RNA-based diseases

New version of gene editing technique corrects molecular mistakes in RNA-based diseases