663 results on '"Waddington, Simon"'
Search Results
2. Genomic investigations of unexplained acute hepatitis in children
3. HIV- 1 lentivirus tethering to the genome is associated with transcription factor binding sites found in genes that favour virus survival
4. Author response: Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome
5. Dysregulated Wnt and NFAT signaling in a Parkinson’s disease LRRK2 G2019S knock-in model
6. GENE THERAPY PREVENTS HEPATIC MITOCHONDRIAL DYSFUNCTION IN MURINE DEOXYGUANOSINE KINASE DEFICIENCY
7. Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease.
8. Rapid and inexpensive purification of adenovirus vectors using an optimised aqueous two-phase technology
9. Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome.
10. mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria
11. Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome
12. Impaired folate 1-carbon metabolism causes formate-preventable hydrocephalus in glycine decarboxylase-deficient mice
13. Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload
14. Systemic gene therapy rescues retinal dysfunction and hearing loss in a model of Norrie disease
15. Ascending vaginal infection in mice induces preterm birth and neonatal morbidity
16. Continual Conscious Bioluminescent Imaging in Freely Moving Mice
17. Ascending Vaginal Infection Using Bioluminescent Bacteria Evokes Intrauterine Inflammation, Preterm Birth, and Neonatal Brain Injury in Pregnant Mice
18. A comparison of intrauterine hemopoietic cell transplantation and lentiviral gene transfer for the correction of severe β-thalassemia in a HbbTh3/+ murine model
19. High-efficiency transduction of spinal cord motor neurons by intrauterine delivery of integration-deficient lentiviral vectors
20. Enhancement of mouse hematopoietic stem/progenitor cell function via transient gene delivery using integration-deficient lentiviral vectors
21. Production of lentiviral vectors using novel, enzymatically produced, linear DNA
22. Fetal gene therapy
23. Human HPSE2gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome
24. Dopamine Transporter Deficiency Syndrome (DTDS): Expanding the Clinical Phenotype and Precision Medicine Approaches
25. Gene Therapy for Dopamine Dyshomeostasis: From Parkinson's to Primary Neurotransmitter Diseases
26. Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors
27. Publisher Correction: In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques
28. Fetal gene therapy.
29. Gene therapy for neurotransmitter‐related disorders.
30. On the Preservation of Evolving Digital Content – The Continuum Approach and Relevant Metadata Models
31. Designing for Inconsistency – The Dependency-Based PERICLES Approach
32. Fetal gene therapy for neurodegenerative disease of infants
33. Dysregulated Wnt and NFAT signaling in a Parkinson’s disease LRRK2 G2019S knock-in model
34. Systemic gene therapy rescues retinal dysfunction and hearing loss in a model of Norrie disease
35. In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques
36. Prime orbit theorems for closed orbits and knots in hyperbolic dynamical systems
37. Microfluidic production of nanogels as alternative triple transfection reagents for the manufacture of adeno-associated virus vectors
38. Specific Lipopolysaccharide Serotypes Induce Differential Maternal and Neonatal Inflammatory Responses in a Murine Model of Preterm Labor
39. Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects
40. Expanding the phenotype in argininosuccinic aciduria: need for new therapies
41. Correction: Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload
42. mRNA therapy restores ureagenesis and corrects glutathione metabolism in argininosuccinic aciduria
43. Bioluminescence Monitoring of Promoter Activity In Vitro and In Vivo
44. Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer
45. Dependency modelling for inconsistency management in Digital Preservation – The PERICLES approach
46. A Broad Overview and Review of CRISPR-Cas Technology and Stem Cells
47. Genomic Investigations of Acute Hepatitis of Unknown Aetiology in Children
48. Mitochondria and Quality Control Defects in a Mouse Model of Gaucher Disease—Links to Parkinson’s Disease
49. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
50. Lentiviral vectors can be used for full-length dystrophin gene therapy
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