35 results on '"Hordeaux, Juliette"'
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2. Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy
3. Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys
4. Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy
5. Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid.
6. Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase
7. Efficacy and Safety of a Krabbe Disease Gene Therapy
8. Development of a novel gene therapy for Fabry disease: Engineered alpha-galactosidase A transgene for improved stability
9. MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates
10. Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology
11. Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
12. Randomized, double-blind trial of F14512, a polyamine-vectorized anticancer drug, compared with etoposide phosphate, in dogs with naturally occurring lymphoma
13. Assessment of adeno-associated virus gene therapies efficacy on acid alpha-glucosidase restoration and glycogen storage correction in cardiac muscle of Pompe disease mice using synchrotron infrared and ultraviolet microspectroscopies
14. The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier
15. Inactivation of the interleukin-22 pathway in the airways of cystic fibrosis patients
16. Standardized Method for Intra-Cisterna Magna Delivery Under Fluoroscopic Guidance in Nonhuman Primates
17. Synchrotron radiation for preclinical research: investigation of tissue chemical change in mouse model of Pompe disease (glycogenosis type II) and assessment of gene therapy efficacy by using Infrared Micro spectroscopy with the synchrotron light
18. Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques
19. Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques
20. The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice
21. Preclinical development of a platform for enzyme therapy in the CNS of MPS I and MPS II patients based on intrathecal AAV delivery
22. Dose escalation study to evaluate safety, tolerability and efficacy of intravenous etoposide phosphate administration in 27 dogs with multicentric lymphoma
23. Synchrotron Infrared Microspectroscopy, an innovative approach to investigate tissue chemical changes in mouse model of Pompe of disease (glycogenosis type II) and to assess efficiency in gene therapy
24. Long-term neurologic and cardiac correction in the Pompe disease mice by intrathecal gene therapy
25. Long-term neurologic correction in the Pompe disease mice by intrathecal gene therapy
26. Selective neuronal targeting by self-complementary AAV9 or AAV10 viaintrathecal delivery in nonhuman primate: application for chronic pain or motorneurons diseases
27. Abstract 4185: Randomized double-blind clinical study of F14512, a new polyamine-vectorized anticancer drug and Etoposide in naturally occurring canine lymphoma
28. Efficient motor neuron and sensory neuron transduction by intrathecal AAVrh10 gene transfer in rats
29. Chronic Traumatic Brain Injury in a Dog
30. Electrophysiological and histopathological changes of the ear and auditory parthways in the mucopolysaccharidosis VII dog
31. Clinical and Pathological study of the hearing function in the mucopolysaccharidosis I Dog American College of veterinary Pathologist
32. Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy.
33. Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
34. Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques.
35. Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques.
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