Whole-body galactose oxidation as a robust functional assay to assess the efficacy of gene-based therapies in a mouse model of Galactosemia.

Authors:
Balakrishnan B; Yan X; McCue MD; Bellagamba O; Guo A and 12 more

Journal:
Mol Ther Methods Clin Dev

Publication Year: 2024

DOI:
10.1016/j.omtm.2024.101191

PMCID:
PMC10863324

PMID:
38352271

View on PubMed View on PMC View Publisher
Journal Information

Full Title: Mol Ther Methods Clin Dev

Abbreviation: Mol Ther Methods Clin Dev

Country: Unknown

Publisher: Unknown

Language: N/A

Publication Details

Subject Category: Medicine, Research & Experimental

Available in Europe PMC: Yes

Available in PMC: Yes

PDF Available: No

Related Papers from Same Journal
AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Fried…
Chang JC; Ryan MR; Stark MC and 16 more (2024) - Transparency Score: 3/6
AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenic…
Doshi BS; Samelson-Jones BJ; Nichols TC and 6 more (2024) - Transparency Score: 3/6
A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mu…
Zhang J; Frabutt DA; Chrzanowski M and 9 more (2024) - Transparency Score: 3/6
B cell focused transient immune suppression protocol for efficient AAV readministration to the live…
Rana J; Herzog RW; Muñoz-Melero M and 9 more (2024) - Transparency Score: 3/6
Continuous manufacturing of lentiviral vectors using a stable producer cell line in a fixed-bed bio…
Stibbs DJ; Silva Couto P; Takeuchi Y and 3 more (2024) - Transparency Score: 3/6
View All Papers from This Journal
Transparency Score
3/6
50.0% Transparent
Transparency Indicators
Click on green indicators to view evidence text
Core Indicators
Data Sharing
Code Sharing
Evidence found in paper:

"Declaration of interests The authors declare the following competing interests: X.Y., F.W., J.T., L.C., R.D., S.C., S.M., Y.W., M.Z., J.S., P.G.V.M., and P.F.F. are employees of Moderna and hold equities from the company."

Evidence found in paper:

"K.L. is supported by the 10.13039/100009633Eunice Kennedy Shriver National Institute of Child Health and Human Development grants R01HD089933 and R21HD104056."

Protocol Registration
Open Access
Paper is freely available to read
Additional Indicators
Replication
Novelty Statement
Assessment Info

Tool: rtransparent

OST Version: N/A

Last Updated: Aug 05, 2025