32 results on '"O'Riordan, Catherine R."'
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2. Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain
3. Combination Brain and Systemic Injections of AAV Provide Maximal Functional and Survival Benefits in the Niemann-Pick Mouse
4. In Vivo Potency Testing of Subretinal rAAV5.hCNGB1 Gene Therapy in the Cngb1 Knockout Mouse Model of Retinitis Pigmentosa
5. CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy
6. Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann--Pick type A disease
7. Targeting the urokinase plasminogen activator receptor enhances gene transfer to human airway epithelia
8. Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes
9. Characteristics of Minimally Oversized Adeno-Associated Virus Vectors Encoding Human Factor VIII Generated Using Producer Cell Lines and Triple Transfection
10. cAMP activates an ATP-permeable pathway in neonatal rat cardiac myocytes
11. 92. Evaluation of Producer Cell Line Platform for Production of Oversized AAV-FVIII Vectors
12. The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo
13. Analytical Ultracentrifugation as an Approach to Characterize Recombinant Adeno-Associated Viral Vectors
14. Strategies to Adapt Adenoviral Vectors for Targeted Delivery
15. Translational Fidelity of Intrathecal Delivery of Self-Complementary AAV9–Survival Motor Neuron 1 for Spinal Muscular Atrophy
16. AAV Vector-Mediated Correction of Brain Pathology in a Mouse Model of Niemann–Pick A Disease
17. Strategies to Adapt Adenoviral Vectors for Targeted Delivery.
18. Targeting a Recombinant Adenovirus Vector to HCC Cells Using a Bifunctional Fab-Antibody Conjugate
19. Scaleable chromatographic purification process for recombinant adeno-associated virus (rAAV)
20. Modification of an Adenoviral Vector with Biologically Selected Peptides: A Novel Strategy for Gene Delivery to Cells of Choice
21. PEGylation of Adenovirus with Retention of Infectivity and Protection from Neutralizing Antibody in Vitro and in Vivo
22. Electrodiffusional ATP movement through the cystic fibrosis transmembrane conductance regulator
23. Purification and Characterization of Recombinant Cystic Fibrosis Transmembrane Conductance Regulator from Chinese Hamster Ovary and Insect Cells
24. Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis
25. Characterization of the oligosaccharide structures associated with the cystic fibrosis transmembrane conductance regulator.
26. 497. Sex and Estrous Cycle Stage Influence the Efficiency of AAV-Mediated Gene Transfer in the Rodent Brain
27. 417. Combination Brain and Systemic Injections of AAV Results in Whole Body Therapy and Extension of Lifespan in the Niemann-Pick Mouse.
28. 110. Performance of Different AAV Serotype Vectors Following Injection into the Deep Cerebellar Nuclei of ASMKO Mouse Brain
29. Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes.
30. The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo.
31. PEGylated adenovirus for targeted gene therapy.
32. Strategies to adapt adenoviral vectors for targeted delivery.
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