Systemic Delivery of AAV-Fdxr Mitigates the Phenotypes of Mitochondrial Disorders in Fdxr Mutant Mice

Li Yang; Jesse Slone; Weiwei Zou; Luis F. Queme; Michael P. Jankowski; Fei Yin; Taosheng Huang

doi:10.1016/j.omtm.2020.05.021

Systemic Delivery of AAV-Fdxr Mitigates the Phenotypes of Mitochondrial Disorders in Fdxr Mutant Mice

Li Yang
, Jesse Slone
, Weiwei Zou
, Luis F. Queme
, Michael P. Jankowski
, Fei Yin
, Taosheng Huang

Pediatrics

Cincinnati Children's Hospital Medical Center
Central South University
Anhui Medical University

Research output: Contribution to journal › Article › peer-review

10 Scopus citations

Abstract

Yang et al. in the Huang group present an effective gene therapy that significantly improves optic and sciatic nerve atrophy, sensory neuropathy, and mitochondrial dysfunction in the FDXR-related mitochondriopathy mouse model. Transgenic products of AAV-PHP.B-Fdxr showed remarkable sustainability. Additionally, its therapeutic effect provides compelling proof for the translation of AAV-PHP.B-Fdxr into clinical applications.

Original language	English
Pages (from-to)	84-97
Number of pages	14
Journal	Molecular Therapy Methods and Clinical Development
Volume	18
DOIs	https://doi.org/10.1016/j.omtm.2020.05.021
State	Published - Sep 11 2020

Access to Document

10.1016/j.omtm.2020.05.021

Cite this

@article{cad23f9794c146b3a8b25bed6f74af15,

title = "Systemic Delivery of AAV-Fdxr Mitigates the Phenotypes of Mitochondrial Disorders in Fdxr Mutant Mice",

abstract = "Yang et al. in the Huang group present an effective gene therapy that significantly improves optic and sciatic nerve atrophy, sensory neuropathy, and mitochondrial dysfunction in the FDXR-related mitochondriopathy mouse model. Transgenic products of AAV-PHP.B-Fdxr showed remarkable sustainability. Additionally, its therapeutic effect provides compelling proof for the translation of AAV-PHP.B-Fdxr into clinical applications.",

author = "Li Yang and Jesse Slone and Weiwei Zou and Queme, \{Luis F.\} and Jankowski, \{Michael P.\} and Fei Yin and Taosheng Huang",

note = "Publisher Copyright: {\textcopyright} 2020 The Authors",

year = "2020",

month = sep,

day = "11",

doi = "10.1016/j.omtm.2020.05.021",

language = "English",

volume = "18",

pages = "84--97",

journal = "Molecular Therapy Methods and Clinical Development",

issn = "2329-0501",

publisher = "Cell Press",

}

TY - JOUR

T1 - Systemic Delivery of AAV-Fdxr Mitigates the Phenotypes of Mitochondrial Disorders in Fdxr Mutant Mice

AU - Yang, Li

AU - Slone, Jesse

AU - Zou, Weiwei

AU - Queme, Luis F.

AU - Jankowski, Michael P.

AU - Yin, Fei

AU - Huang, Taosheng

PY - 2020/9/11

Y1 - 2020/9/11

N2 - Yang et al. in the Huang group present an effective gene therapy that significantly improves optic and sciatic nerve atrophy, sensory neuropathy, and mitochondrial dysfunction in the FDXR-related mitochondriopathy mouse model. Transgenic products of AAV-PHP.B-Fdxr showed remarkable sustainability. Additionally, its therapeutic effect provides compelling proof for the translation of AAV-PHP.B-Fdxr into clinical applications.

AB - Yang et al. in the Huang group present an effective gene therapy that significantly improves optic and sciatic nerve atrophy, sensory neuropathy, and mitochondrial dysfunction in the FDXR-related mitochondriopathy mouse model. Transgenic products of AAV-PHP.B-Fdxr showed remarkable sustainability. Additionally, its therapeutic effect provides compelling proof for the translation of AAV-PHP.B-Fdxr into clinical applications.

UR - https://www.scopus.com/pages/publications/85086590837

U2 - 10.1016/j.omtm.2020.05.021

DO - 10.1016/j.omtm.2020.05.021

M3 - Article

AN - SCOPUS:85086590837

SN - 2329-0501

VL - 18

SP - 84

EP - 97

JO - Molecular Therapy Methods and Clinical Development

JF - Molecular Therapy Methods and Clinical Development

ER -

Systemic Delivery of AAV-Fdxr Mitigates the Phenotypes of Mitochondrial Disorders in Fdxr Mutant Mice

Abstract

Access to Document

Other files and links

Fingerprint

Cite this