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63 results on '"Edoardo Nusco"'

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1. Genome editing without nucleases confers proliferative advantage to edited hepatocytes and corrects Wilson disease

2. TFEB and TFE3 drive kidney cystogenesis and tumorigenesis

3. Liver‐directed gene therapy for ornithine aminotransferase deficiency

4. Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice

5. O-GlcNAcylation enhances CPS1 catalytic efficiency for ammonia and promotes ureagenesis

6. Liver gene therapy with intein‐mediated F8 trans‐splicing corrects mouse haemophilia A

7. Therapeutic homology-independent targeted integration in retina and liver

8. Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease

9. Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors

10. Induction of Autophagy Promotes Clearance of RHOP23H Aggregates and Protects From Retinal Degeneration

11. Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA

12. Retinal Degeneration in MPS-IIIA Mouse Model

13. AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease

14. Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI

15. A highly secreted sulphamidase engineered to cross the blood‐brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA

16. Modelling TFE renal cell carcinoma in mice reveals a critical role of WNT signaling

17. Disease rescue and increased lifespan in a model of cardiomyopathy and muscular dystrophy by combined AAV treatments.

18. Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors

19. CHOP and c-JUN up-regulate the mutant Z α1-antitrypsin, exacerbating its aggregation and liver proteotoxicity

20. A substrate-specific mTORC1 pathway underlies Birt–Hogg–Dubé syndrome

21. The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease

22. microRNAs as biomarkers in Pompe disease

23. Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease

24. Enhanced Version of Human Sulfamidase Significantly Ameliorates CNS Pathology When Delivered to the MPS-IIIA Mice by AAV-Mediated Intra-CSF Injection

25. CHOP and c-JUN up-regulate the mutant Z α

26. CHOP-c-JUN complex plays a critical role in liver proteotoxicity induced by mutant Z alpha-1 antitrypsin

27. Light‐responsive microRNA miR‐211 targets Ezrin to modulate lysosomal biogenesis and retinal cell clearance

28. Pyruvate dehydrogenase complex and lactate dehydrogenase are targets for therapy of acute liver failure

29. Enhancement of hepatic autophagy increases ureagenesis and protects against hyperammonemia

30. Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI

31. >)Transcriptional activation of RagD GTPase controls mTORC1 and promotes cancer growth

32. Retinal Degeneration in MPS-IIIA Mouse Model

33. mTORC1 hyperactivation arrests bone growth in lysosomal storage disorders by suppressing autophagy

35. Author response: Modelling TFE renal cell carcinoma in mice reveals a critical role of WNT signaling

36. Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease

37. Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy

39. SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies

40. 280. Combination of Low-Dose Gene Therapy and Monthly Enzyme Replacement Therapy Improves the Phenotype of a Mouse Model of Lysosomal Storage Disease

41. FGF signalling regulates bone growth through autophagy

42. Tbx1 regulates brain vascularization

43. Phenylbutyrate Therapy for Pyruvate Dehydrogenase Complex Deficiency and Lactic Acidosis

44. A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA

45. Improved efficacy and reduced toxicity by ultrasound-guided intrahepatic injections of helper-dependent adenoviral vector in Gunn rats

46. Modelling TFE renal cell carcinoma in mice reveals a critical role of WNT signaling

47. Sustained reduction of hyperbilirubinemia in Gunn rats after adeno-associated virus-mediated gene transfer of bilirubin UDP-glucuronosyltransferase isozyme 1A1 to skeletal muscle

48. Impaired parkin-mediated mitochondrial targeting to autophagosomes differentially contributes to tissue pathology in lysosomal storage diseases

49. Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder

50. Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB

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