Use of an adeno‐associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model-Reference-Cited by-同舟云学术

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Use of an adeno‐associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model

Published:2021-08-19 Issue:6 Volume:44 Page:1369-1381
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:Jrnl of Inher Metab Disea

Author:

Kaiser Robert A.¹²^ORCID,Weber Nicholas D.³,Trigueros‐Motos Laia³,Allen Kari L.²,Martinez Michael⁴,Cao William²,VanLith Caitlin J.²,Hillin Lori G.²,Douar Anne⁵,González‐Aseguinolaza Gloria³⁶⁷^ORCID,Aldabe Rafael⁶,Lillegard Joseph B.¹²⁸

Affiliation:

1. Children's Hospitals and Clinics of Minnesota Minneapolis Minnesota USA

2. Department of Surgery Mayo Clinic Rochester Minnesota USA

3. Vivet Therapeutics S.L. Pamplona Spain

4. Department of Molecular and Medical Genetics Oregon Health & Science University Portland Oregon USA

5. Vivet Therapeutics S.A.S. Paris France

6. Division of Gene Therapy and Regulation of Gene Expression CIMA Universidad de Navarra Pamplona Spain

7. Instituto de Investigación Sanitaria de Navarra (IdISNA) Pamplona Spain

8. Pediatric Surgical Associates Minneapolis Minnesota USA

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/jimd.12392

Reference28 articles.

1. Phenylketonuria

2. Monogenic traits are not simple: lessons from phenylketonuria

3. Executive function in early-treated phenylketonuria: Profile and underlying mechanisms☆☆☆

4. Long-term correction of hyperphenylalaninemia by AAV-mediated gene transfer leads to behavioral recovery in phenylketonuria mice

5. Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer

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1. Current Advances and Material Innovations in the Search for Novel Treatments of Phenylketonuria;Advanced Healthcare Materials;2024-05-31

2. Characterizing the mechanism of action for mRNA therapeutics for the treatment of propionic acidemia, methylmalonic acidemia, and phenylketonuria;Nature Communications;2024-05-07

3. Treatment of a genetic liver disease in mice through transient prime editor expression;2024-01-24

4. Topoisomerase Inhibitors Increase Episomal DNA Expression by Inducing the Integration of Episomal DNA in Hepatic Cells;Pharmaceutics;2023-10-13

5. In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans;Molecular Therapy - Methods & Clinical Development;2023-09

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