Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia-Reference-Cited by-同舟云学术

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Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia

Published:2023-07-01 Issue:13-14 Volume:34 Page:605-615
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Munoz-Zuluaga Carlos¹,Gertz Monica¹,Yost-Bido Melissa¹,Greco Alessandria¹,Gorman Nicholas¹,Chen Alvin¹,Kooner Vikrum¹,Rosenberg Jonathan B.¹,De Bishnu P.¹,Kaminsky Stephen M.¹,Borczuk Alain²,Ricart Arbona Rodolfo J.³,Martin Heather R.³,Monette Sebastien³,Khanna Richie⁴,Barth Jay A.⁴,Crystal Ronald G.¹,Sondhi Dolan¹

Affiliation:

1. Department of Genetic Medicine, New York, New York, USA.

2. Department of Pathology, Weill Cornell Medicine, New York, New York, USA.

3. Center for Comparative Medicine and Pathology, Memorial Sloan Kettering Cancer Center, Weill Cornell Medicine, New York, New York, USA.

4. LEXEO Therapeutics, New York, New York, USA.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2023.020

Reference31 articles.

1. Mortality in Friedreich Ataxia

2. Studies of human, mouse and yeast homologues indicate a mitochondrial function for frataxin

3. The Structure and Function of Frataxin

4. Frataxin: a protein in search for a function

5. Fixing frataxin: ‘ironing out’ the metabolic defect in Friedreich's ataxia

Cited by 6 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. New and Emerging Drug and Gene Therapies for Friedreich Ataxia;CNS Drugs;2024-08-08

2. Expression and processing of mature human frataxin after gene therapy in mice;Scientific Reports;2024-04-10

3. AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich’s ataxia;Molecular Therapy - Methods & Clinical Development;2024-03

4. Hereditary Ataxias: From Bench to Clinic, Where Do We Stand?;Cells;2024-02-09

5. Expression and processing of mature human frataxin after gene therapy in mice;2023-12-28

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