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