Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy

Nalinda B. Wasala; Yongping Yue; William Lostal; Lakmini P. Wasala; Nandita Niranjan; Roger J. Hajjar; Gopal J. Babu; Dongsheng Duan

doi:10.1016/j.ymthe.2019.12.011

Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy

Nalinda B. Wasala, Yongping Yue, William Lostal, Lakmini P. Wasala, Nandita Niranjan, Roger J. Hajjar, Gopal J. Babu, Dongsheng Duan

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

34 Scopus citations

Abstract

Increased cytosolic calcium level is a pathogenic hallmark in Duchenne muscular dystrophy. The sarcoplasmic/endoplasmic reticulum (SR) calcium ATPase (SERCA) pump transfers cytosolic calcium to the SR. Wasala et al. show that AAV-mediated overexpression of SERCA2a at 3 months of age resulted in lifelong prevention of cardiomyopathy in a mouse model.

Original language	English
Pages (from-to)	845-854
Number of pages	10
Journal	Molecular Therapy
Volume	28
Issue number	3
DOIs	https://doi.org/10.1016/j.ymthe.2019.12.011
State	Published - 4 Mar 2020
Externally published	Yes

Keywords

AAV
DMD
Duchenne muscular dystrophy
SERCA
calcium
dilated cardiomyopathy
dystrophin
gene therapy
mdx
sarcoplasmic/endoplasmic reticulum

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title = "Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy",

abstract = "Increased cytosolic calcium level is a pathogenic hallmark in Duchenne muscular dystrophy. The sarcoplasmic/endoplasmic reticulum (SR) calcium ATPase (SERCA) pump transfers cytosolic calcium to the SR. Wasala et al. show that AAV-mediated overexpression of SERCA2a at 3 months of age resulted in lifelong prevention of cardiomyopathy in a mouse model.",

keywords = "AAV, DMD, Duchenne muscular dystrophy, SERCA, calcium, dilated cardiomyopathy, dystrophin, gene therapy, mdx, sarcoplasmic/endoplasmic reticulum",

author = "Wasala, {Nalinda B.} and Yongping Yue and William Lostal and Wasala, {Lakmini P.} and Nandita Niranjan and Hajjar, {Roger J.} and Babu, {Gopal J.} and Dongsheng Duan",

note = "Publisher Copyright: {\textcopyright} 2020 The American Society of Gene and Cell Therapy",

year = "2020",

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doi = "10.1016/j.ymthe.2019.12.011",

language = "English",

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journal = "Molecular Therapy",

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T1 - Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy

AU - Wasala, Nalinda B.

AU - Yue, Yongping

AU - Lostal, William

AU - Wasala, Lakmini P.

AU - Niranjan, Nandita

AU - Hajjar, Roger J.

AU - Babu, Gopal J.

AU - Duan, Dongsheng

PY - 2020/3/4

Y1 - 2020/3/4

N2 - Increased cytosolic calcium level is a pathogenic hallmark in Duchenne muscular dystrophy. The sarcoplasmic/endoplasmic reticulum (SR) calcium ATPase (SERCA) pump transfers cytosolic calcium to the SR. Wasala et al. show that AAV-mediated overexpression of SERCA2a at 3 months of age resulted in lifelong prevention of cardiomyopathy in a mouse model.

AB - Increased cytosolic calcium level is a pathogenic hallmark in Duchenne muscular dystrophy. The sarcoplasmic/endoplasmic reticulum (SR) calcium ATPase (SERCA) pump transfers cytosolic calcium to the SR. Wasala et al. show that AAV-mediated overexpression of SERCA2a at 3 months of age resulted in lifelong prevention of cardiomyopathy in a mouse model.

KW - AAV

KW - DMD

KW - Duchenne muscular dystrophy

KW - SERCA

KW - calcium

KW - dilated cardiomyopathy

KW - dystrophin

KW - gene therapy

KW - mdx

KW - sarcoplasmic/endoplasmic reticulum

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SN - 1525-0016

VL - 28

SP - 845

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JO - Molecular Therapy

JF - Molecular Therapy

IS - 3

ER -

Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy

Abstract

Keywords

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