Search

Your search keyword '"Venditti, Charles P."' showing total 249 results

Search Constraints

Start Over You searched for: Author "Venditti, Charles P." Remove constraint Author: "Venditti, Charles P." Language english Remove constraint Language: english
249 results on '"Venditti, Charles P."'

Search Results

10. 1-13C-propionate breath testing as a surrogate endpoint to assess efficacy of liver-directed therapies in methylmalonic acidemia (MMA)

18. Chronic kidney disease in propionic acidemia

20. Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

25. Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia.

26. List of Contributors

27. Contributors

28. MECHANISMS OF DISEASE

31. Targeting proximal tubule mitochondrial dysfunction attenuates the renal disease of methylmalonic acidemia

33. Biomarkers to predict disease progression and therapeutic response in isolated methylmalonic acidemia.

37. Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy

39. Methylmalonic acidemia: a megamitochondrial disorder affecting the kidney

41. New insights into the pathophysiology of methylmalonic acidemia.

47. Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience.

50. Characterization of the null murine sodium/myo-inositol cotransporter 1 (Smit1 or Slc5a3) phenotype: Myo-inositol rescue is independent of expression of its cognate mitochondrial ribosomal protein subunit 6 (Mrps6) gene and of phosphatidylinositol levels in neonatal brain

Catalog

Books, media, physical & digital resources