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.
Preclinical study demonstrates efficacy of gene therapy in treatment of Duchenne muscular dystrophy

Preclinical study demonstrates efficacy of gene therapy in treatment of Duchenne muscular dystrophy

Genetic therapy for two inherited neurologic diseases moves closer to human clinical trials

Genetic therapy for two inherited neurologic diseases moves closer to human clinical trials

Developing software for cell and gene therapy supply chain tracking

Developing software for cell and gene therapy supply chain tracking

'Organic gene therapy' could be effective treatment approach for serious blood disorders

'Organic gene therapy' could be effective treatment approach for serious blood disorders

MGH researchers identify novel mechanism behind resistance to angiogenesis inhibitors

MGH researchers identify novel mechanism behind resistance to angiogenesis inhibitors

Scientists uncover new gene therapy treatment routes for motor neurone disease

Scientists uncover new gene therapy treatment routes for motor neurone disease

FDA panel recommends new breakthrough gene therapy for B-cell acute lymphoblastic leukemia (ALL)

FDA panel recommends new breakthrough gene therapy for B-cell acute lymphoblastic leukemia (ALL)

FDA advisory committee recommends Novartis' CAR-T therapy for young leukemia patients

FDA advisory committee recommends Novartis' CAR-T therapy for young leukemia patients

New partnership to advance novel airway-delivered gene therapy for treating pulmonary hypertension

New partnership to advance novel airway-delivered gene therapy for treating pulmonary hypertension

Synthetic compound that reads mitochondrial DNA could lead to treatments for nerve, muscle diseases

Synthetic compound that reads mitochondrial DNA could lead to treatments for nerve, muscle diseases

Loss of smell linked to weight loss in mice despite same food intake, study reveals

Loss of smell linked to weight loss in mice despite same food intake, study reveals

Sense of smell may play key role in how the body deals with calories

Sense of smell may play key role in how the body deals with calories

Researchers uncover novel mechanism of beta-blockers in preventing progression of heart failure

Researchers uncover novel mechanism of beta-blockers in preventing progression of heart failure

Study sheds light on how key protein allows hearing to work

Study sheds light on how key protein allows hearing to work

International team to set how gene therapies in hemophilia should be measured for effectiveness

International team to set how gene therapies in hemophilia should be measured for effectiveness

Altered virus could help more patients to become eligible for human gene therapy trials

Altered virus could help more patients to become eligible for human gene therapy trials

Study reveals role of mast cells in severe forms of dengue infection

Study reveals role of mast cells in severe forms of dengue infection

Researchers find long-lasting benefits of gene therapy in dog model of myotubular myopathy

Researchers find long-lasting benefits of gene therapy in dog model of myotubular myopathy

New immunotherapy sends multiple myeloma into remission

New immunotherapy sends multiple myeloma into remission

New breakthrough offers hope for allergy sufferers

New breakthrough offers hope for allergy sufferers