Sentences with phrase «aav gene therapy vectors»

AAV gene therapy vectors are emerging as the gene transfer vehicle with high potential for use in the CNS as they transduce post mitotic cells that mediate the sustained, long term gene expression required in chronic progressive diseases.

Not exact matches

As the central nervous system is also a target of AAV vectors, Hélène Puccio and Patrick Aubourg's teams are investigating whether a similar approach using gene therapy could be as effective for the spinal cord and cerebellum as it is for the heart.

(Adeno - associated virus or AAV has been approved in Europe as a vector for gene therapy, and is being explored in the United States for clinical trials.)

They then stitched the genes for these immunoadhesins into an adeno - associated virus (AAV), a «vector» used in human gene therapy experiments to deliver foreign DNA into the body's cells.

Efficient AAV Vector Production System: Towards Gene Therapy For Duchenne Muscular Dystrophy.