Correction of a knock-in mouse model of acrodysostosis with gene therapy using a rAAV9-CAG-human PRKAR1A vector
Author:
Funder
INSERM
CEA-Neuratris, Therapy Design Consulting
Publisher
Springer Science and Business Media LLC
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.nature.com/articles/s41434-021-00286-2.pdf
Reference33 articles.
1. Silve C, Le Stunff C, Motte E, Gunes Y, Linglart A, Clauser E. Acrodysostosis syndromes. BoneKEy Rep. 2012;225:1–7.
2. Linglart A, Menguy C, Couvineau A, Auzan C, Gunes Y, Cancel M, et al. Recurrent PRKAR1A mutation in acrodysostosis with hormone resistance. N Engl J Med. 2011;364:2218–26.
3. Rhayem Y, Le Stunff C, Khalek WA, Auzan C, Bertherat J, Linglart A, et al. Functional characterization of PRKAR1A mutations reveals a unique molecular mechanism causing acrodysostosis but multiple mechanisms causing Carney complex. J Biol Chem. 2015;290:27816–28.
4. Le Stunff C, Tilotta F, Sadoine J, Le Denmat D, Briet C, Motte E, et al. Knock-In of the Recurrent R368X Mutation of PRKAR1A that Represses cAMP‐Dependent Protein Kinase A Activation: a Model of Type 1 Acrodysostosis. J Bone Mineral Res. 2017;32:333–46.
5. Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019;18:358–78.
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1. Current Status and Prospects of Viral Vector-Based Gene Therapy to Treat Kidney Diseases;Human Gene Therapy;2024-03-01
2. Phosphodiesterase 4D activity in acrodysostosis-associated neural pathology: too much or too little?;Brain Communications;2024
3. Cell and gene therapy for kidney disease;Nature Reviews Nephrology;2023-03-27
4. Gene therapy for kidney disease: targeting cystinuria;Current Opinion in Nephrology & Hypertension;2021-12-15
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