AAV ‐mediated expression of galactose‐1‐phosphate uridyltransferase corrects defects of galactose metabolism in classic galactosemia patient fibroblasts-Reference-Cited by-同舟云学术

AAV ‐mediated expression of galactose‐1‐phosphate uridyltransferase corrects defects of galactose metabolism in classic galactosemia patient fibroblasts

Published:2022-01-23 Issue: Volume: Page:
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J of Inher Metab Disea

Author:

Brophy Megan L.¹^ORCID,Stansfield John C.²,Ahn Youngwook³,Cheng Seng H.¹,Murphy John E.¹,Bell Robert D.¹

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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