40 results on '"Anguela, Xavier M"'
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2. Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec
3. Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A
4. Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice
5. Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B
6. IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies
7. Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates
8. In vivo genome editing of the albumin locus as a platform for protein replacement therapy
9. Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
10. Liver Production of Sulfamidase Reverses Peripheral and Ameliorates CNS Pathology in Mucopolysaccharidosis IIIA Mice
11. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
12. In vivo genome editing restores haemostasis in a mouse model of haemophilia
13. Nonviral-Mediated Hepatic Expression of IGF-I Increases Treg Levels and Suppresses Autoimmune Diabetes in Mice
14. Oracle or false prophet? Can we predict AAV efficacy based on preexisting antibody titers?
15. Reversal of Type 1 Diabetes by Engineering a Glucose Sensor in Skeletal Muscle
16. Preclinical development of , an investigational liver-directed AAV gene therapy for the treatment of Pompe disease
17. Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year
18. Entering the Modern Era of Gene Therapy
19. A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds
20. Preclinical development of SPK-3006, an investigational liver-directed AAV gene therapy for the treatment of Pompe disease
21. Robust ZFN-mediated genome editing in adult hemophilic mice
22. Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression
23. An edible switch for gene therapy
24. 52. Therapeutic Factor VIII Expression After AAV Delivery in Non-Human Primates
25. In Vivo Genome Editing in Neonatal Mouse Liver Preferentially Utilizes Homology Directed Repair
26. Adeno-Associated Viral Vector Delivery of Optimized Human Factor VIII Achieves Therapeutic Factor VIII Levels in Non-Human Primates
27. Adeno-associated viral vectors for the treatment of hemophilia
28. Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response
29. Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence
30. Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a
31. ZFN Mediated Targeting Of Albumin “Safe Harbor” Results In Therapeutic Levels Of Human Factor VIII In a Mouse Model Of Hemophilia A
32. Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys
33. Adeno-associated viral vectors for the treatment of hemophilia.
34. In Vivo Genome Editing of Liver Albumin for Therapeutic Gene Expression: Rescue of Hemophilic Mice Via Integration of Factor 9
35. A Novel Strategy to Circumvent Pre-Existing Humoral Immunity to AAV
36. Robust Factor IX Expression Following ZFN-Mediated Genome Editing in An Adult Mouse Model of Hemophilia B
37. In vivo genome editing restores haemostasis in a mouse model of haemophilia.
38. In VivoGenome Editing in Neonatal Mouse Liver Preferentially Utilizes Homology Directed Repair
39. In VivoGenome Editing of Liver Albumin for Therapeutic Gene Expression: Rescue of Hemophilic Mice Via Integration of Factor 9
40. 901. Recovery from Type 1 Diabetes by Insulin and Glucokinase Expression in Skeletal Muscle
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