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First-in-human pilot trial for new sickle cell disease gene therapy approach proves quicker, more efficient than industry average

Researchers from Boston Children's Hospital have developed a new gene therapy approach that collects enough stem cells in a single hospital admission, improving turnaround time for genetically altering and infusing cells. The approach has shown long-term stability and safety, with patients not experiencing any adverse effects.

SourceBoston Children's Hospital·JournalBlood·DateAug 25, 2026

3D genome architecture influences SCID-X1 gene therapy success

Researchers identified the integration site pattern of lentiviral gene therapies in patient cells, shedding light on treatment safety and efficacy. The study found that integration near nuclear pores is associated with improved safety and effectiveness, avoiding oncogene regions where earlier technologies failed.

SourceSt. Jude Children's Research Hospital·JournalScience Advances·TypeRandomized controlled/clinical trial·DateOct 6, 2023

Bold new therapy delivery method shows initial promise as treatment for Duchenne muscular dystrophy

A new therapy delivery method, using modified viruses engineered with fusogens Myomaker and Myomerger, shows promise as a treatment for Duchenne muscular dystrophy. The vector can deliver a vital gene needed for muscle function to cells, potentially providing a lifelong supply of the missing gene.

SourceCincinnati Children's Hospital Medical Center·JournalCell·TypeExperimental study·DateApr 27, 2023