469 results on '"Alexander, Ian E"'
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2. AAV-delivered hepato-adrenal cooperativity in steroidogenesis: Implications for gene therapy for congenital adrenal hyperplasia
3. Development of CNS tropic AAV1-like variants with reduced liver-targeting following systemic administration in mice
4. Liver-specific adiponectin gene therapy suppresses microglial NLRP3-inflammasome activation for treating Alzheimer’s disease
5. Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation
6. Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes
7. Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy
8. Gene Therapy for Paediatric Homozygous Familial Hypercholesterolaemia
9. In Search of Adeno-Associated Virus Vectors With Enhanced Cardiac Tropism for Gene Therapy
10. Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution
11. Gain-of-function factor H–related 5 protein impairs glomerular complement regulation resulting in kidney damage
12. Stable transduction of the neonatal mouse liver using a hybrid rAAV/sleeping beauty transposon gene delivery system.
13. Gene therapy clinical trials worldwide to 2023—an update.
14. Liver-specific deletion of miR-181ab1 reduces liver tumour progression via upregulation of CBX7
15. The Balance of Stromal BMP Signaling Mediated by GREM1 and ISLR Drives Colorectal Carcinogenesis
16. Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes
17. Genome editing in the human liver: Progress and translational considerations
18. The diagnostic utility of genome sequencing in a pediatric cohort with suspected mitochondrial disease
19. The implementation of newborn screening for spinal muscular atrophy: the Australian experience
20. Gene therapy for urea cycle defects: An update from historical perspectives to future prospects.
21. Harnessing the power of whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation
22. Human Connexin40 Mutations Slow Conduction and Increase Propensity for Atrial Fibrillation
23. Thymocyte self-renewal and oncogenic risk in immunodeficient mouse models: relevance for human gene therapy clinical trials targeting haematopoietic stem cell populations?
24. The self-peptide repertoire plays a critical role in transplant tolerance induction
25. Gene therapy for urea cycle defects: An update from historical perspectives to future prospects
26. Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors
27. Recapitulation of Skewed X-Inactivation in Female Ornithine Transcarbamylase-Deficient Primary Human Hepatocytes in the FRG Mouse: A Novel System for Developing Epigenetic Therapies.
28. AAV-Mediated Gene Delivery to the Mouse Liver
29. AAV-mediated gene delivery of the calreticulin anti-angiogenic domain inhibits ocular neovascularization
30. Future Directions for Adrenal Insufficiency: Cellular Transplantation and Genetic Therapies
31. Persistent Expression of Human Clotting Factor IX from Mouse Liver after Intravenous Injection of Adeno-Associated Virus Vectors
32. Intrabiliary infusion of naked DNA vectors targets periportal hepatocytes in mice
33. Assessment of pre-clinical liver models based on their ability to predict the liver-tropism of AAV vectors
34. Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects
35. Mitochondrial Respiratory Chain Hepatopathies: Role of Liver Transplantation. A Case Series of Five Patients
36. Future directions for adrenal insufficiency: cellular transplantation and genetic therapies
37. Performance of Cardiotropic rAAV Vectors Is Dependent on Production Method
38. Isling: A Tool for Detecting Integration of Wild-Type Viruses and Clinical Vectors
39. AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression
40. A bioinformatic pipeline for simulating viral integration data
41. Antigen expression level threshold tunes the fate of CD8 T cells during primary hepatic immune responses
42. Blockade of HMGB1 Attenuates Diabetic Nephropathy in Mice
43. Onasemnogene abeparvovec for the treatment of spinal muscular atrophy
44. Conversion of the Liver into a Biofactory for DNaseI Using Adeno-Associated Virus Vector Gene Transfer Reduces Neutrophil Extracellular Traps in a Model of Systemic Lupus Erythematosus
45. Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8
46. The Potential of AAV-Mediated Gene Targeting for Gene and Cell Therapy Applications
47. Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy
48. Angiotensin Converting Enzyme‐2 Therapy Improves Liver Fibrosis and Glycemic Control in Diabetic Mice With Fatty Liver
49. Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys
50. Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
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