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

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

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

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

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

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

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

31. Retinal Degeneration in MPS-IIIA Mouse Model

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

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

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

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

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

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

38. FGF signalling regulates bone growth through autophagy

39. Tbx1 regulates brain vascularization

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

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

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

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

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

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

46. Systemic inflammation and neurodegeneration in a mouse model of multiple sulfatase deficiency

47. Disease Rescue and Increased Lifespan in a Model of Cardiomyopathy and Muscular Dystrophy by Combined AAV Treatments

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

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