Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease von Hordeaux, J, Dubreil, L, Robveille, C, Deniaud, J, Pascal, Q, Dequéant, B, Pailloux, J, Lagalice, L, Ledevin, M, Babarit, C, Costiou, P, Jamme, F, Fusellier, M, Mallem, Y, Ciron, C, Huchet, C, Caillaud, C, Colle, M-A

Volltext

Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats von Hordeaux, J, Dubreil, L, Deniaud, J, Iacobelli, F, Moreau, S, Ledevin, M, Le Guiner, C, Blouin, V, Le Duff, J, Mendes-Madeira, A, Rolling, F, Cherel, Y, Moullier, P, Colle, M-A

Volltext

Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy von Rojsajjakul, Teerapat, Hordeaux, Juliette J., Choudhury, Gourav R., Hinderer, Christian J., Mesaros, Clementina, Wilson, James M., Blair, Ian A.

Volltext

Colorectal Hamartomatous Polyposis and Ganglioneuromatosis in a Dog von Bemelmans, I., Küry, S., Albaric, O., Hordeaux, J., Bertrand, L., Nguyen, F., Abadie, J.

Volltext

Histopathologic Changes of the Ear in Canine Models of Mucopolysaccharidosis Types I and VII von Hordeaux, J., Deniaud, J., Bemelmans, I., Bertrand, L., Moreau, S., Amiaud, J., Wyers, M., Cherel, Y., Colle, M.-A.

Volltext

Chronic Traumatic Brain Injury in a Dog von Laurent, S., Thibaud, J.L., Hordeaux, J., Reyes-Gomez, E., Delisle, F., Blot, S., Colle, M.A.

Volltext

Auditory and Nervous System Pathology in a Mouse Model of Pompe Disease von Franzoso, F.D., Ciron, C., Hordeaux, J., Bagarie, C., Deniaud, J., Colle, M.A.

Volltext

Efficient Central Nervous System Transduction by Intracisternal AAV10 Gene Transfer in Rats von Hordeaux, J., Moreau, S., Dubreil, L., Deniaud, J., Iacobelli, F., Joussemet, B., Le Guiner, C., Moullier, P., Chérel, Y., Colle, M.-A.

Volltext

Additional file 1: of Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease von J. Hordeaux, L. Dubreil, C. Robveille, J. Deniaud, Q. Pascal, B. DequĂŠant, J. Pailloux, L. Lagalice, M. Ledevin, C. Babarit, P. Costiou, F. Jamme, M. Fusellier, Y. Mallem, C. Ciron, C. Huchet, C. Caillaud, M-A Colle

Volltext bestellen

The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier von Hordeaux, Juliette, Yuan, Yuan, Clark, Peter M., Wang, Qiang, Martino, R. Alexander, Sims, Joshua J., Bell, Peter, Raymond, Angela, Stanford, William L., Wilson, James M.

Volltext

Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid von Rosenberg, Jonathan B, Fung, Edward K, Dyke, Jonathan P, De, Bishnu P, Lou, Howard, Kelly, James M, Reejhsinghani, Layla, Ricart Arbona, Rodolfo J, Sondhi, Dolan, Kaminsky, Stephen M, Cartier, Nathalie, Hinderer, Christian, Hordeaux, Juliette, Wilson, James M, Ballon, Douglas J, Crystal, Ronald G

Volltext