Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study.

Authors:
Ryckman AE; Deschenes NM; Quinville BM; Osmon KJL; Mitchell M and 3 more

Journal:
Mol Ther Methods Clin Dev

Publication Year: 2023

DOI:
10.1016/j.omtm.2023.101168

PMCID:
PMC10777117

PMID:
38205442

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

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"Declaration of interests The authors declare no competing interests."

Evidence found in paper:

"Funding for this research was provided by 10.13039/100012515Cure Tay-Sachs Foundation and 10.13039/501100009056GlycoNet."

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Last Updated: Aug 05, 2025