Collaborative effort by investing in the development of curative therapies that can be delivered safely, effectively and affordably in low-resource settings
Collaboration aims to develop affordable gene therapies
News
Gene Therapy
Added to gene therapy, compound enable faster and effective treatments
Research team finds that a nontoxic molecule closely related to resveratrol can overcome barriers to delivering gene therapy into stem cells
Nanocapsules deliver gene-editing payload
Structures are alternatives to viral vectors for gene therapy using the CRISPR-Cas9 system
Gene editing enables researchers to correct mutation in muscle stem cells
Duchenne muscular dystrophy (DMD) is a rare but devastating genetic disorder that causes muscle loss and physical impairments. Researchers have shown that CRISPR may provide the means for lifelong correction of the genetic mutation responsible for the disorder
Stem cell researchers reactivate ‘back-up genes’ in the lab
Researchers have unravelled parts of a mechanism that may one day help to treat Rett syndrome and other genetic disorders linked to the X chromosome.
Gene therapy helps functional recovery after stroke
A new gene therapy turns glial cells into neurons, repairing damage that results from stroke and significantly improving motor function
Targeting a subset of stem cells shows lasting gene editing in blood cells
A new Science Translational Medicine paper is first to report how editing a portion of stem cells with CRISPR/Cas9 is sufficient for long-term reactivation of therapeutic hemoglobin
Slug, a stem cell regulator, promotes repair of DNA damage
Data point towards Slug acting as a safeguard against breast tissue aging, as it promotes both stem cell activity and efficient DNA damage repair
Scientists developing way to help premature babies breathe easier
Study points to possible cell therapy for Bronchopulmonary Dysplasia