144 results on '"McCarty, Douglas M."'
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2. Realizing the promise of gene therapy through collaboration and partnering : Pfizer’s view
3. Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery
4. Serum global metabolomics profiling reveals profound metabolic impairments in patients with MPS IIIA and MPS IIIB
5. Topics in AAV integration come front and center at ASGCT AAV Integration Roundtable
6. Differential distribution of heparan sulfate glycoforms and elevated expression of heparan sulfate biosynthetic enzyme genes in the brain of mucopolysaccharidosis IIIB mice
7. Integration of adeno-associated virus (AAV) and recombinant AAV vectors
8. Analysis of the interaction between adeno-associated virus and heparan sulfate using atomic force microscopy
9. Mucopolysaccharidosis IIIB, a lysosomal storage disease, triggers a pathogenic CNS autoimmune response
10. Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats
11. Targeting Root Cause by Systemic scAAV9-hIDS Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice
12. Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy
13. Effective Depletion of Pre-existing Anti-AAV Antibodies Requires Broad Immune Targeting
14. Systemic gene transfer of scAAV9.U1a.hSGSH for MPS IIIA: tolerability and preliminary evidence for a biochemical effect
15. Global metabolomics profiling reveals profound metabolic impairments in patients with mucopolysaccharidosis type III
16. Crossing the blood–brain-barrier with viral vectors
17. 698. Effective Depletion of Pre-Existing Anti-AAV Antibodies Requires Broad Immune Targeting
18. 162. Functional Correction of Mucopolysaccharidosis I in Adult Mice by a Systemic rAAV9-IDUA Gene Delivery
19. 352. Functional Benefits of Systemic rAAV9-hIDS Gene Delivery in MPS II Mouse Model
20. Functional benefits of systemic rAAV9-hids gene delivery in MPS II mouse model
21. Functional correction of mucopolysaccharidosis I in adult mice by a systemic rAAV9-IDUA gene delivery
22. Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery
23. A GLP-compliant toxicology and biodistribution study: systemic delivery of a rAAV9 vector for the treatment of mucopolysaccharidosis IIIB
24. High-resolution gel and hybridization analysis of plant mitochondrial RNA
25. Sequence and transcriptional analysis of a chloroplast insert in the mitochondrial genome ofZea mays
26. 78. An IND-Enabling GLP-Toxicology and Biodistribution Study Assessing Systemic rAAV9-hNAGLU Gene Delivery for Treating MPS IIIB: Genotype- and Sex-Specific Dose-Limiting Acute Liver Toxicity in Male Wild Type C57BL/6 Mice
27. 172. Correction of Profound Metabolic Impairments in MPS IIIB Mice by a Systemic rAAV9-hNAGLU Gene Delivery
28. Broad Functional Correction of Molecular Impairments by Systemic Delivery of scAAVrh74-hSGSH Gene Delivery in MPS IIIA Mice
29. Blood Genome-Wide Transcriptional Profiles Reflect Broad Molecular Impairments and Strong Blood-Brain Links in Alzheimer's Disease
30. Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating Mucopolysaccharidosis IIIB: Toxicology, Biodistribution, and Immunological Assessments in Primates
31. Gene Therapy for Neurodevelopmental Disorders
32. Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating MPS IIIB: Toxicology, Bio-distribution and Immunological Assessments in Primates
33. Functional correction and reversal of MPS IIIA in a mouse model by an intravenous hSGSH gene delivery using a self-complementary AAV9 vector
34. Correction of broad molecular impairments in a mouse model of MPS IIIA by systemic rAAVrh74-hSGSH gene delivery
35. Amyloidosis, Synucleinopathy, and Prion Encephalopathy in a Neuropathic Lysosomal Storage Disease: The CNS-Biomarker Potential of Peripheral Blood
36. Improved brain expression of iduronate sulfatase in the MPS II mouse after intravenous delivery of a self-complimentary adeno-associated viral (AAV) vector
37. Patterns of scAAV Vector Insertion Associated With Oncogenic Events in a Mouse Model for Genotoxicity
38. Peripheral Nervous System Neuropathology and Progressive Sensory Impairments in a Mouse Model of Mucopolysaccharidosis IIIB
39. Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood–Brain Barrier Gene Delivery
40. Differential Effects of DNA Double-Strand Break Repair Pathways on Single-Strand and Self-Complementary Adeno-Associated Virus Vector Genomes
41. Mucopolysaccharidosis IIIB, a lysosomal storage disease, triggers a pathogenic CNS autoimmune response
42. Restoration of central nervous system α-N-acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno-associated viral type 2 vector delivery
43. Innate and adaptive immune activation in the brain of MPS IIIB mouse model
44. Self-complementary AAV Vectors; Advances and Applications
45. Blood Genome-Wide Transcriptional Profiles Reflect Broad Molecular Impairments and Strong Blood-Brain Links in Alzheimer's Disease.
46. Host Cell DNA Repair Pathways in Adeno-Associated Viral Genome Processing
47. 494. Optimizing the Mannitol-Facilitated CNS Entry of Peripherally-Delivered AAV2 Vector
48. Intrathecal Long-Term Gene Expression by Self-Complementary Adeno-Associated Virus type 1 Suitable for Chronic Pain Studies in Rats
49. Effects of Adeno-Associated Virus DNA Hairpin Structure on Recombination
50. Adeno-associated virus serotypes 1 to 5 mediated tumor cell directed gene transfer and improvement of transduction efficiency
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