Search Results - "Hordeaux, J"

Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease by 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

“…Pompe disease is a lysosomal storage disorder caused by acid-α-glucosidase (GAA) deficiency, leading to glycogen storage. The disease manifests as a fatal…”
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Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy by Rojsajjakul, Teerapat, Hordeaux, Juliette J., Choudhury, Gourav R., Hinderer, Christian J., Mesaros, Clementina, Wilson, James M., Blair, Ian A.

“…Deficiency in human mature frataxin (hFXN-M) protein is responsible for the devastating neurodegenerative and cardiodegenerative disease of Friedreich’s ataxia…”
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Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats by 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

“…Intracerebral administration of recombinant adeno-associated vector (AAV) has been performed in several clinical trials. However, delivery into the brain…”
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Colorectal Hamartomatous Polyposis and Ganglioneuromatosis in a Dog by Bemelmans, I., Küry, S., Albaric, O., Hordeaux, J., Bertrand, L., Nguyen, F., Abadie, J.

“…A 5-month-old female Great Dane puppy was treated for hematochezia, tenesmus, and rectal prolapse by resection of a 10-cm-long segment of colon and rectum…”
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Histopathologic Changes of the Ear in Canine Models of Mucopolysaccharidosis Types I and VII by Hordeaux, J., Deniaud, J., Bemelmans, I., Bertrand, L., Moreau, S., Amiaud, J., Wyers, M., Cherel, Y., Colle, M.-A.

“…Mucopolysaccharidosis (MPS) types I and VII are inborn errors of metabolism caused by mutation of enzymes involved in glycosaminoglycan catabolism, which leads…”
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Chronic Traumatic Brain Injury in a Dog by Laurent, S., Thibaud, J.L., Hordeaux, J., Reyes-Gomez, E., Delisle, F., Blot, S., Colle, M.A.

“…Chronic traumatic brain injury is rare in man and has not been previously documented in dogs. This report describes a 2-year-old female American Staffordshire…”
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Auditory and Nervous System Pathology in a Mouse Model of Pompe Disease by Franzoso, F.D., Ciron, C., Hordeaux, J., Bagarie, C., Deniaud, J., Colle, M.A.

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

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