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.
Amish people live longer due to mutation in blood clotting gene

Amish people live longer due to mutation in blood clotting gene

Gene editing within a living person for the first time

Gene editing within a living person for the first time

Study finds genetic treatment for inherited retinal disorder

Study finds genetic treatment for inherited retinal disorder

New gene therapy for blindness may soon be reality

New gene therapy for blindness may soon be reality

Complete new skin from transgenic stem cells for boy with rare genetic disease

Complete new skin from transgenic stem cells for boy with rare genetic disease

New gene therapy trial for X-linked myotubular myopathy offers hope to children

New gene therapy trial for X-linked myotubular myopathy offers hope to children

Study solves critical piece of deafness puzzle by identifying long non-coding RNAs

Study solves critical piece of deafness puzzle by identifying long non-coding RNAs

Conference to focus on preventing and reversing consequences of sickle cell disease

Conference to focus on preventing and reversing consequences of sickle cell disease

Gene therapy prevents age-related learning and memory problems in mice

Gene therapy prevents age-related learning and memory problems in mice

Research advance may lead to preservation of hearing in people with Usher syndrome type III

Research advance may lead to preservation of hearing in people with Usher syndrome type III

VICC selected to administer FDA-approved CAR T therapy for treatment of lymphoma patients

VICC selected to administer FDA-approved CAR T therapy for treatment of lymphoma patients

Scientists receive critical funding to study treatments for three deadly cancers

Scientists receive critical funding to study treatments for three deadly cancers

Cascade of costs could push new gene therapy above $1 million per patient

Cascade of costs could push new gene therapy above $1 million per patient

FDA advisory committee vote 16-0 for first even gene therapy for rare blindness

FDA advisory committee vote 16-0 for first even gene therapy for rare blindness

MIT researchers develop new technique to screen for genes that protect against diseases

MIT researchers develop new technique to screen for genes that protect against diseases

New microcapsules represent promising vectors for application of genome-editing tools

New microcapsules represent promising vectors for application of genome-editing tools

CGT Catapult’s iPSC line licensed to Universal Cells for creating universal donor cell line

CGT Catapult’s iPSC line licensed to Universal Cells for creating universal donor cell line

Moms of children with rare genetic illness push for wider newborn screening

Moms of children with rare genetic illness push for wider newborn screening

Gene therapy effectively stabilizes progression of cerebral adrenoleukodystrophy, trial reveals

Gene therapy effectively stabilizes progression of cerebral adrenoleukodystrophy, trial reveals

New gene therapy to treat cancer in children, young adults offers hopes and challenges

New gene therapy to treat cancer in children, young adults offers hopes and challenges