Sentences with phrase «therapeutic gene delivery»
These alternate strategies include using small - molecule drugs that affect RNA metabolism or protein stability, as well as administering modified viruses for therapeutic gene delivery (see «Getting a fix on SMA»).
http://www.smafoundation.org/ Not exact matches
To use viruses as delivery vehicles for gene therapy, researchers take all the harmful and replicative genes out of the virus and put in the therapeutic genes they want to deliver.
This molecule brings a revolutionary technology platform for genetic engineering in vertebrates, including gene discovery in model species and for therapeutic transgene delivery for possible human applications.
Jessica Tucker, program director of Drug and Gene Delivery and Devices at the National Institute of Biomedical Imaging and Bioengineering, which is part of the National Institutes of Health, said the approach outlined in the study has the ability to combine both imaging and therapeutic applications in a single platform, which has been difficult to achieve, especially in an organic, and therefore biocompatible, vehicle.
The first step in both direct and cell - based gene therapy is packaging a therapeutic transgene into a delivery vehicle (e.g., a viral vector), followed by expansion of its host cell lines to produce high - enough vector concentrations.
Duan also provided significant expertise from a long history of work on gene therapy for neuromuscular disorders.To use viruses as delivery vehicles for gene therapy, researchers take all the harmful and replicative genes out of the virus and put in the therapeutic genes they want to deliver.