AAV ‐mediated expression of galactose‐1‐phosphate uridyltransferase corrects defects of galactose metabolism in classic galactosemia patient fibroblasts
Author:
Affiliation:
1. Rare Disease Research Unit Pfizer, Inc. Cambridge Massachusetts USA
2. Early Clinical Development Pfizer, Inc. Cambridge Massachusetts USA
3. Target Sciences Pfizer, Inc. Cambridge Massachusetts USA
Publisher
Wiley
Subject
Genetics (clinical),Genetics
Link
https://onlinelibrary.wiley.com/doi/pdf/10.1002/jimd.12468
Reference54 articles.
1. Hereditary galactosemia
2. Galactosemia, a Single Gene Disorder With Epigenetic Consequences
3. The unfolded protein response has a protective role in yeast models of classic galactosemia
4. Downregulation of Insulin-Like Growth Factor-1 via Nitric Oxide Production in a Hypergalactosemic Model of Neonate Skin Fibroblast Cultures
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