95 results on '"Byrne, Barry J."'
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2. In memoriam: Kenneth I. Berns, MD, PhD (1938–2024)
3. Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency
4. Nicholas Muzyczka, PhD
5. Secondary hemophagocytic lymphohistiocytosis following Zolgensma therapy: An evolving story on the innate response to systemic gene therapy
6. Advanced therapeutic strategy for hereditary neuromuscular diseases
7. Scratching the surface of RGD-directed AAV capsid engineering
8. Mavis Agbandje-McKenna’s lifelong commitment to teaching and research
9. Current Clinical Applications of In Vivo Gene Therapy with AAVs
10. Considerations for Systemic Use of Gene Therapy
11. Neuromuscular Gene Therapy: Catching the Wave of Positive Early Studies Is Expected to Crest in Product Approvals
12. Obituary: Kenneth I. Berns, MD, PhD (1938–2024)
13. Safety First: Perspective on Patient-Centered Development of AAV Gene Therapy Products
14. 382. Correction of Autophagic Dysregulation in Pompe Disease Following Gene Therapy
15. 472. Evaluation of Re-Administration of a Recombinant Adeno-Associated Vector Expressing Acid-Alpha-Glucosidase (rAAV9-DES-hGAA) in Pompe Disease: Preclinical to Clinical Planning
16. 180. The Role of the Pyruvate Dehydrogenase Kinase 4 (PDK4) Gene Mutation in the Development of Dilated Cardiomyopathy in Dobermanpinschers and Potential Application of Cardiac Gene Therapy
17. Benefits of Neuronal Preferential Systemic Gene Therapy for Neurotransmitter Deficiency
18. 368. Simultaneous Delivery of Dual AAV9 Vectors Induces Immune Tolerance and Correction of Pompe Disease Pathology
19. C-11. An Update on Gene Therapy for the Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
20. 201. Neuron-Specific Systemic Gene Therapy for Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
21. 401. AAV Therapy Attenuates Respiratory Dysfunction and Glycogen Accumulation in a Murine Model of Glycogen Storage Disease Type II
22. 187. AAV Delivery of Tafazzin for the Correction of Mitochondrial Dysfunction in Barth Syndrome Using IPS Differentiated Cardiomyocytes
23. 399. Early Correction of Neuromuscular Deficits in Pompe Disease Is Required for Restoration of Strength Following Gene Therapy
24. Charting a Clear Path: The ASGCT Standardized Pathways Conference
25. Sustained Correction of Motoneuron Histopathology Following Intramuscular Delivery of AAV in Pompe Mice
26. Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease
27. Pathway for Approval of a Gene Therapy Orphan Product: Treading New Ground
28. Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases
29. Signs of Progress in Gene Therapy for Muscular Dystrophy Also Warrant Caution
30. Spinal Delivery of AAV Vector Restores Enzyme Activity and Increases Ventilation in Pompe Mice
31. AAV Vectors for Cardiac Gene Transfer: Experimental Tools and Clinical Opportunities
32. AAV6-Mediated Gene Silencing fALS Short
33. Gel-mediated Delivery of AAV1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease
34. Long-term Skeletal Muscle Protection After Gene Transfer in a Mouse Model of LGMD-2D
35. Physiological Correction of Pompe Disease by Systemic Delivery of Adeno-associated Virus Serotype 1 Vectors
36. 892. Neural Deficits Contribute to Respiratory Insufficiency in Pompe Disease: A Therapeutic Approach with AAV1
37. 575. Correction of Ventilation in Glycogen Storage Disease Type II Mice after Gel-Mediated Delivery of Adeno-Associated Virus Serotype 1 Vectors
38. 709. Adeno-Associated Virus Delivery of siRNAs Leads to a Reduction in Phospholamban Levels
39. 728. Safety and Bioactivity of rAAV2-hAAT in alpha-1 Antitrypsin-Deficient Patients in a Phase I Clinical Trial
40. 28. rAAV2/9 Mediated Gene Delivery of Acid α-Glucosidase Corrects the Cardiac Phenotype in a Mouse Model of Pompe Disease
41. Successful Production of Pseudotyped rAAV Vectors Using a Modified Baculovirus Expression System
42. Herpesvirus-based infectious titering of recombinant adeno-associated viral vectors
43. A New Method for Recombinant Adeno-associated Virus Vector Delivery to Murine Diaphragm
44. Improved Method of Recombinant AAV2 Delivery for Systemic Targeted Gene Therapy
45. Correction of the Enzymatic and Functional Deficits in a Model of Pompe Disease Using Adeno-associated Virus Vectors
46. Long-term efficacy and safety of eladocageneexuparvovec in patients with AADC deficiency
47. Current Clinical Applications of In VivoGene Therapy with AAVs
48. Advanced therapeutic strategy for hereditary neuromuscular diseases
49. 514. Effect of DNA-PKcs on AAV Replication
50. 909. Sustained Correction of Glycogen Storage Disease Type II by rAAV1 Vector-Mediated Gene Therapy
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