Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

Haiyan Zhou; Jinhong Meng; Alberto Malerba; Francesco Catapano; Palittiya Sintusek; Susan Jarmin; Lucy Feng; Ngoc Lu-Nguyen; Lianwen Sun; Virginie Mariot; Julie Dumonceaux; Jennifer E. Morgan; Paul Gissen; George Dickson; Francesco Muntoni

doi:10.1002/jcsm.12542

Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

Haiyan Zhou, Jinhong Meng, Alberto Malerba, Francesco Catapano, Palittiya Sintusek, Susan Jarmin, Lucy Feng, Ngoc Lu-Nguyen, Lianwen Sun, Virginie Mariot, Julie Dumonceaux, Jennifer E. Morgan, Paul Gissen, George Dickson, Francesco Muntoni

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

Original language	English
Pages (from-to)	768-782
Number of pages	15
Journal	Journal of Cachexia, Sarcopenia and Muscle
Volume	11
Issue number	3
Early online date	7 Feb 2020
DOIs	https://doi.org/10.1002/jcsm.12542
Publication status	Published - Jun 2020

Keywords

Adeno-associated virus
Antisense oligonucleotide
Combinatorial therapy
Myostatin inhibition
Myostatin propeptide
Spinal muscular atrophy
Survival of motor neuron 1 gene

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10.1002/jcsm.12542Licence: CC BY

Cite this

Zhou, H., Meng, J., Malerba, A., Catapano, F., Sintusek, P., Jarmin, S., Feng, L., Lu-Nguyen, N., Sun, L., Mariot, V., Dumonceaux, J., Morgan, J. E., Gissen, P., Dickson, G., & Muntoni, F. (2020). Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy. Journal of Cachexia, Sarcopenia and Muscle, 11(3), 768-782. https://doi.org/10.1002/jcsm.12542

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title = "Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy",

keywords = "Adeno-associated virus, Antisense oligonucleotide, Combinatorial therapy, Myostatin inhibition, Myostatin propeptide, Spinal muscular atrophy, Survival of motor neuron 1 gene",

author = "Haiyan Zhou and Jinhong Meng and Alberto Malerba and Francesco Catapano and Palittiya Sintusek and Susan Jarmin and Lucy Feng and Ngoc Lu-Nguyen and Lianwen Sun and Virginie Mariot and Julie Dumonceaux and Morgan, {Jennifer E.} and Paul Gissen and George Dickson and Francesco Muntoni",

year = "2020",

month = jun,

doi = "10.1002/jcsm.12542",

language = "English",

volume = "11",

pages = "768--782",

journal = "Journal of Cachexia, Sarcopenia and Muscle",

issn = "2190-5991",

publisher = "Wiley",

number = "3",

}

Zhou, H, Meng, J, Malerba, A, Catapano, F, Sintusek, P, Jarmin, S, Feng, L, Lu-Nguyen, N, Sun, L, Mariot, V, Dumonceaux, J, Morgan, JE, Gissen, P, Dickson, G & Muntoni, F 2020, 'Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy', Journal of Cachexia, Sarcopenia and Muscle, vol. 11, no. 3, pp. 768-782. https://doi.org/10.1002/jcsm.12542

TY - JOUR

T1 - Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

AU - Zhou, Haiyan

AU - Meng, Jinhong

AU - Malerba, Alberto

AU - Catapano, Francesco

AU - Sintusek, Palittiya

AU - Jarmin, Susan

AU - Feng, Lucy

AU - Lu-Nguyen, Ngoc

AU - Sun, Lianwen

AU - Mariot, Virginie

AU - Dumonceaux, Julie

AU - Morgan, Jennifer E.

AU - Gissen, Paul

AU - Dickson, George

AU - Muntoni, Francesco

PY - 2020/6

Y1 - 2020/6

KW - Adeno-associated virus

KW - Antisense oligonucleotide

KW - Combinatorial therapy

KW - Myostatin inhibition

KW - Myostatin propeptide

KW - Spinal muscular atrophy

KW - Survival of motor neuron 1 gene

UR - http://www.scopus.com/inward/record.url?scp=85079134341&partnerID=8YFLogxK

U2 - 10.1002/jcsm.12542

DO - 10.1002/jcsm.12542

M3 - Article

C2 - 32031328

AN - SCOPUS:85079134341

SN - 2190-5991

VL - 11

SP - 768

EP - 782

JO - Journal of Cachexia, Sarcopenia and Muscle

JF - Journal of Cachexia, Sarcopenia and Muscle

IS - 3

ER -

Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

Keywords

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Destructive Exon Skipping of Myostatin mRNA with and without Open Reading Frame Rescue of Dystrophin

Combination Antisense Treatment for Duchenne Muscular Dystrophy: Open Reading Frame Rescue of Dystrophin in conjunction with Destructive Exon Skipping of Myostatin mRNA

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Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

Keywords

Access to Document

Other files and links

Projects

Destructive Exon Skipping of Myostatin mRNA with and without Open Reading Frame Rescue of Dystrophin

Combination Antisense Treatment for Duchenne Muscular Dystrophy: Open Reading Frame Rescue of Dystrophin in conjunction with Destructive Exon Skipping of Myostatin mRNA

Cite this