99 results on '"Byrne, Barry J."'
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2. B cell focused transient immune suppression protocol for efficient AAV readministration to the liver
3. Switching treatment to cipaglucosidase alfa plus miglustat positively affects motor function and quality of life in patients with late-onset Pompe disease
4. Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL
5. Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy
6. Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation
7. Optogenetic activation of the tongue in spontaneously breathing mice
8. Long-term follow-up of cipaglucosidase alfa/miglustat in ambulatory patients with Pompe disease: An open-label phase I/II study (ATB200-02)
9. Nicholas Muzyczka, PhD
10. Polysomnography findings in children with spinal muscular atrophy after onasemnogene-abeparvovec
11. Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy
12. Secondary hemophagocytic lymphohistiocytosis following Zolgensma therapy: An evolving story on the innate response to systemic gene therapy
13. Comparison of highly pure rAAV9 vector stocks produced in suspension by PEI transfection or HSV infection reveals striking quantitative and qualitative differences
14. Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency
15. Advanced therapeutic strategy for hereditary neuromuscular diseases
16. Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial
17. Scratching the surface of RGD-directed AAV capsid engineering
18. NaCl and KCl mediate log increase in AAV vector particles and infectious titers in a specific/timely manner with the HSV platform
19. Mavis Agbandje-McKenna’s lifelong commitment to teaching and research
20. Current Clinical Applications of In Vivo Gene Therapy with AAVs
21. Considerations for Systemic Use of Gene Therapy
22. Arginine kinetics are altered in a pilot sample of adolescents and young adults with Barth syndrome
23. Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease
24. AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice
25. Neuromuscular Gene Therapy: Catching the Wave of Positive Early Studies Is Expected to Crest in Product Approvals
26. AAV9-TAZ Gene Replacement Ameliorates Cardiac TMT Proteomic Profiles in a Mouse Model of Barth Syndrome
27. Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study
28. Safety and efficacy of advanced and targeted acid α-glucosidase (AT-GAA) (ATB200/AT2221) in ERT-switch nonambulatory patients with Pompe disease: preliminary results from the ATB200-02 trial
29. Preliminary patient-reported outcomes and safety of advanced and targeted acid α-glucosidase (AT-GAA (ATB200/AT2221) in patients with Pompe disease from the ATB200-02 trial
30. First-in-human study of advanced and targeted acid α-glucosidase (AT-GAA) (ATB200/AT2221) in patients with Pompe disease: preliminary functional assessment results from the ATB200-02 trial
31. Safety First: Perspective on Patient-Centered Development of AAV Gene Therapy Products
32. Compensatory responses to unexpected inspiratory loads are distinct from clinical tests of respiratory capacity
33. Long-term study of growth and development outcomes in patients with infantile-onset Pompe disease receiving alglucosidase alfa: Safety data update
34. Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial
35. 13C/31P MRS Metabolic Biomarkers of Disease Progression and Response to AAV Delivery of hGAA in a Mouse Model of Pompe Disease
36. Immune Modulatory Cell Therapy for Hemophilia B Based on CD20-Targeted Lentiviral Gene Transfer to Primary B Cells
37. Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticity
38. An injectable capillary-like microstructured alginate hydrogel improves left ventricular function after myocardial infarction in rats
39. Generation and characterization of anti-Adeno-associated virus serotype 8 (AAV8) and anti-AAV9 monoclonal antibodies
40. Neuropathology in respiratory-related motoneurons in young Pompe (Gaa) mice
41. 382. Correction of Autophagic Dysregulation in Pompe Disease Following Gene Therapy
42. 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
43. 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
44. Altered activation of the diaphragm in late-onset Pompe disease
45. Targeted approaches to induce immune tolerance for Pompe disease therapy
46. A scalable method for the production of high-titer and high-quality adeno-associated type 9 vectors using the HSV platform
47. Benefits of Neuronal Preferential Systemic Gene Therapy for Neurotransmitter Deficiency
48. BAFF blockade prevents anti-drug antibody formation in a mouse model of Pompe disease
49. 368. Simultaneous Delivery of Dual AAV9 Vectors Induces Immune Tolerance and Correction of Pompe Disease Pathology
50. C-11. An Update on Gene Therapy for the Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
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