BRAINCAV is an FP7-funded collaborative research project that focuses on the development and assessment of a gene transfer vector to target the brain. As suggested by recent clinical results, brain gene transfer offers substantial potential, yet brings unique obstacles - in particular the need to address feasibility, efficacy and safety. BRAINCAV's foundation is based on the potential of canine adenovirus type 2 (CAV-2) vectors to preferentially transduce neurons, undergo efficient targeting via axonal transport and provide long-term expression. BRAINCAV is a translational project that spans basic research through pre-clinical model feasibility, efficacy and safety. The BRAINCAV partners developed cell lines & purification schemes, improved production protocols, assayed the transcriptional response of vector-transduced neurons in vitro and in vivo, characterized the mechanism of receptor engagement and axonal transport, and assayed biodistribution and interaction with brain fluids and cells. To provide a proof-of-principle of the effectiveness of CAV-2 vectors, we also tackled mucopolysaccharidosis type VII, a global, orphan disease commonly affecting children, and Parkinson's disease, a focal degeneration of dopaminergic neurones commonly affecting aged population. A collection of BRAINCAV posters describing our latest results will be presented. For the second consecutive year, a 1500 € “BRAINCAV prize” will be awarded to a selected author of poster for gene transfer to the brain.
BRAINCAV: a nonhuman adenovirus vector for gene transfer to the brain / P., Alves Marques; A., Baker; A. A., Bosch; M., Carrondo; M., Ej Kremer; R., Luquin; Saggio, Isabella; J., Schwarz; G., Schiavo; J. M., Verdier. - In: HUMAN GENE THERAPY. - ISSN 1043-0342. - STAMPA. - 23:(2012), pp. A154-A154. (Intervento presentato al convegno Collaborative Congress of the European-Society-of-Gene-and-Cell-Therapy/French-Society-of-Cell-and-Gene-Therapy tenutosi a Versailles, FRANCE nel OCT 25-29, 2012).
BRAINCAV: a nonhuman adenovirus vector for gene transfer to the brain
SAGGIO, Isabella;
2012
Abstract
BRAINCAV is an FP7-funded collaborative research project that focuses on the development and assessment of a gene transfer vector to target the brain. As suggested by recent clinical results, brain gene transfer offers substantial potential, yet brings unique obstacles - in particular the need to address feasibility, efficacy and safety. BRAINCAV's foundation is based on the potential of canine adenovirus type 2 (CAV-2) vectors to preferentially transduce neurons, undergo efficient targeting via axonal transport and provide long-term expression. BRAINCAV is a translational project that spans basic research through pre-clinical model feasibility, efficacy and safety. The BRAINCAV partners developed cell lines & purification schemes, improved production protocols, assayed the transcriptional response of vector-transduced neurons in vitro and in vivo, characterized the mechanism of receptor engagement and axonal transport, and assayed biodistribution and interaction with brain fluids and cells. To provide a proof-of-principle of the effectiveness of CAV-2 vectors, we also tackled mucopolysaccharidosis type VII, a global, orphan disease commonly affecting children, and Parkinson's disease, a focal degeneration of dopaminergic neurones commonly affecting aged population. A collection of BRAINCAV posters describing our latest results will be presented. For the second consecutive year, a 1500 € “BRAINCAV prize” will be awarded to a selected author of poster for gene transfer to the brain.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
