129 results on '"LeBowitz, Jonathan H."'
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2. Predicting disease severity in metachromatic leukodystrophy using protein activity and a patient phenotype matrix
3. Identifying therapeutic drug targets using bidirectional effect genes
4. Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B
5. Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB
6. Glycosylation-Independent Targeting Enhances Enzyme Delivery to Lysosomes and Decreases Storage in Mucopolysaccharidosis Type VII Mice
7. Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction
8. Additional file 4 of Predicting disease severity in metachromatic leukodystrophy using protein activity and a patient phenotype matrix
9. Additional file 12 of Predicting disease severity in metachromatic leukodystrophy using protein activity and a patient phenotype matrix
10. Haploinsufficiency underlies the neurodevelopmental consequences ofSLC6A1/GAT-1 variants
11. Initiation of DNA Replication on Single-Stranded DNA Templates Catalyzed by Purified Replication Proteins of Bacteriophage λ and Escherichia coli
12. Development of a Stable Leishmania Expression Vector and Application to the Study of Parasite Surface Antigen Genes
13. The dnaK Protein of Escherichia coli Possesses an ATPase and Autophosphorylating Activity and is Essential in an in vitro DNA Replication System
14. Initiation of Bacteriophage λ DNA Replication in vitro with Purified λ Replication Proteins
15. A Glycosylphosphatidylinositol (GPI)-Negative Phenotype Produced in Leishmania major by GPI Phospholipase C from Trypanosoma brucei: Topography of Two GPI Pathways
16. Cloning of a Lymphoid-Specific cDNA Encoding a Protein Binding the Regulatory Octamer DNA Motif
17. Correction: Intermittent enzyme replacement therapy prevents Neu1 deficiency
18. Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice
19. Intermittent enzyme replacement therapy with recombinant human β-galactosidase prevents neuraminidase 1 deficiency
20. A Breach in the Blood-Brain Barrier
21. Identifying therapeutic drug targets for rare and common forms of short stature
22. Inhibition of endogenous miR‐23a/miR‐377 in CHO cells enhances difficult‐to‐express recombinant lysosomal sulfatase activity
23. Characterization of glycan substrates accumulating in GM1 Gangliosidosis
24. Chapter 4: Transfection Experiments with Leishmania
25. Assessment of predicted enzymatic activity of α‐ N ‐acetylglucosaminidase variants of unknown significance for CAGI 2016
26. Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B
27. BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis
28. BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis
29. Utilizing ExAC to assess the hidden contribution of variants of unknown significance to Sanfilippo Type B incidence
30. Initiation of DNA Synthesis on Single-Stranded DNA Templates in Vitro Promoted by the Bacteriophage λ O and P Replication Proteins
31. Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice
32. Cellular Uptake and Delivery of Myeloperoxidase to Lysosomes Promote Lipofuscin Degradation and Lysosomal Stress in Retinal Cells
33. Immunohistochemical analysis of mannose 6-phosphate/insulin-like growth factor 2 receptor in murine wild-type and mucopolysaccharidosis IIIB mutant central nervous system vasculature and implications for trans-blood brain barrier (BBB) transport
34. Histologic characterization of the progression of central nervous system pathology in the mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo syndrome type B) mouse model and bio-distribution and efficacy of the intracerebroventricular enzyme replacement therapy, BMN 250
35. Utilizing activity assays and population-wide allele frequencies to assess the contribution of novel mutations in NAGLU to MPS IIIB incidence
36. Glycosylation independent lysosomal targeting of alpha-n-acetylglucosaminidase confers highly efficient enzyme uptake into critical cellular targets of disease pathogenesis in mucopolysaccharidosis type IIIB
37. Evaluation of myeloperoxidase as a targeted enzymatic approach for the elimination of retinal A2E in Stargardt disease
38. Time- and dose-dependent normalization of pathological lysosomal storage and biochemistry in the mucopolysaccharidosis ΙΙΙΒ (MPS ΙΙΙΒ, Sanfilippo syndrome type Β) mouse model by intracerebroventricular enzyme replacement therapy with ΒΜΝ 250, a ΝAGLU-ΙGF2 fusion pro
39. Engineering of a recombinant NAGLU fusion protein with insulin-like growth factor 2 leads to improved cellular uptake via a glycosylation-independent lysosomal targeting pathway
40. BMN 701-mediated receptor redistribution is responsible for increased uptake
41. Glycosylation-independent Lysosomal Targeting of Acid α-Glucosidase Enhances Muscle Glycogen Clearance in Pompe Mice
42. Coordinate regulation of a family of promastigote-enriched mRNAs by the 3′UTR PRE element in Leishmania mexicana
43. The two cytoplasmic dynein-2 isoforms in Leishmania mexicana perform separate functions
44. Relationships between the major kinetoplastid paraflagellar rod proteins: a consolidating nomenclature
45. A Negative Regulatory Element Controls mRNA Abundance of the Leishmania mexicana Paraflagellar Rod Gene PFR2
46. Unravelling the kinetoplastid paraflagellar rod
47. The promoter of the human interleukin-2 gene contains two octamer-binding sites and is partially activated by the expression of Oct-2
48. Stage-specific expression of the Leishmania mexicana paraflagellar rod protein PFR-2
49. Leishmania mexicana promastigotes induce cytotoxic T lymphocytes in vivo that do not recognize infected macrophages
50. Thymidine kinase as a negative selectable marker in Leishmania major
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