Differential myofiber-type transduction preference of adeno-associated virus serotypes 6 and 9

Riaz, Muhammad Bilal; Raz, Yotam; Moloney, Elizabeth B.; Putten, Maaike van; Krom, Yvonne D.; Maarel, Silvère M. van der; Verhaagen, Joost; Raz, Vered

doi:10.1186/s13395-015-0064-4

Cited by 35 publications

(36 citation statements)

References 34 publications

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“…To avoid an in vivo immune response, adeno-associated virus (AAV)-9, which is specific to skeletal muscle with broad myofiberspecific tropism, was used (Kotterman & Schaffer, 2014;Riaz et al, 2015). The recombinant mGPDH-encoding AAV9 and the control vectors (AAV9-GFP) were generated by GENE Company.…”

Section: Aav Gene Transfermentioning

confidence: 99%

Mitochondrial glycerol 3‐phosphate dehydrogenase promotes skeletal muscle regeneration

Liu

Zheng

et al. 2018

EMBO Mol Med

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While adult mammalian skeletal muscle is stable due to its post‐mitotic nature, muscle regeneration is still essential throughout life for maintaining functional fitness. During certain diseases, such as the modern pandemics of obesity and diabetes, the regeneration process becomes impaired, which leads to the loss of muscle function and contributes to the global burden of these diseases. However, the underlying mechanisms of the impairment are not well defined. Here, we identify mGPDH as a critical regulator of skeletal muscle regeneration. Specifically, it regulates myogenic markers and myoblast differentiation by controlling mitochondrial biogenesis via CaMKKβ/AMPK. mGPDH−/− attenuated skeletal muscle regeneration in vitro and in vivo, while mGPDH overexpression ameliorated dystrophic pathology in mdx mice. Moreover, in patients and animal models of obesity and diabetes, mGPDH expression in skeletal muscle was reduced, further suggesting a direct correlation between its abundance and muscular regeneration capability. Rescuing mGPDH expression in obese and diabetic mice led to a significant improvement in their muscle regeneration. Our study provides a potential therapeutic target for skeletal muscle regeneration impairment during obesity and diabetes.

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Section: Aav Gene Transfermentioning

confidence: 99%

Mitochondrial glycerol 3‐phosphate dehydrogenase promotes skeletal muscle regeneration

Liu

Zheng

et al. 2018

EMBO Mol Med

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“…In this context, serotype AAV9 shows high cardiac‐ and skeletal muscle tropism (Asokan et al ., 2012; Katwal et al ., 2013; Riaz et al ., 2015), maximizing transgene expression. Nevertheless, since systemic application of AAV9 vectors also lead to the transduction of other organ systems, tissue‐specific promoters are essential to minimize off‐target effects or overall toxicity of the transgene (Wang et al ., 2008).…”

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confidence: 99%

A compact unc45b‐promoter drives muscle‐specific expression in zebrafish and mouse

Rudeck

Etard

Khan

et al. 2016

Genesis

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Summary: Gene therapeutic approaches to cure genetic diseases require tools to express the rescuing gene exclusively within the affected tissues. Viruses are often chosen as gene transfer vehicles but they have limited capacity for genetic information to be carried and transduced. In addition, to avoid off‐target effects the therapeutic gene should be driven by a tissue‐specific promoter in order to ensure expression in the target organs, tissues, or cell populations. The larger the promoter, the less space will be left for the respective gene. Thus, there is a need for small but tissue‐specific promoters. Here, we describe a compact unc45b promoter fragment of 195 bp that retains the ability to drive gene expression exclusively in skeletal and cardiac muscle in zebrafish and mouse. Remarkably, the described unc45b promoter fragment not only drives muscle‐specific expression but presents heat‐shock inducibility, allowing a temporal and spatial quantity control of (trans)gene expression. Here, we demonstrate that the transgenic expression of the smyd1b gene driven by the unc45b promoter fragment is able to rescue the embryonically lethal heart and skeletal muscle defects in smyd1b‐deficient flatline mutant zebrafish. Our findings demonstrate that the described muscle‐specific unc45b promoter fragment might be a valuable tool for the development of genetic therapies in patients suffering from myopathies. genesis 54:431–438, 2016. © 2016 The Authors. Genesis Published by Wiley Periodicals, Inc.

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“…Male, 5‐week‐old C57BL/6J mice (Jackson laboratories, Sacramento, CA, USA) were acclimatized for 1 week and standard conditions as detailed in Riaz et al .,25 under the Animal Research: Reporting of In Vivo Experiments guidelines. An animal research protocol [#13113] was approved by the Institutional Animal Ethical Committee, Leiden University Medical Center, Leiden, the Netherlands.…”

Section: Methodsmentioning

confidence: 99%

“…The immunofluorescence procedure was carried out as detailed in Riaz et al 14, 25. Images were captured with the DM5500 microscope (Leica) using the LAS AF software version 2.3.6.…”

Section: Methodsmentioning

confidence: 99%

Proteasomal activity‐based probes mark protein homeostasis in muscles

Raz¹,

Raz²,

Soriano

et al. 2017

J cachexia sarcopenia muscle

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BackgroundProtein homeostasis, primarily regulated by the ubiquitin–proteasome system is crucial for proper function of cells. In tissues of post‐mitotic cells, the impaired ubiquitin–proteasome system is found in a wide range of neuromuscular disorders. Activity‐based probes (ABPs) measure proteasomal proteolytic subunits and can be used to report protein homeostasis. Despite the crucial role of the proteasome in neuromuscular pathologies, ABPs were not employed in muscle cells and tissues, and measurement of proteasomal activity was carried out in vitro using low‐throughput procedures.MethodsWe screened six ABPs for specific application in muscle cell culture using high throughput call‐based imaging procedures. We then determined an in situ proteasomal activity in myofibers of muscle cryosections.ResultsWe demonstrate that LWA300, a pan‐reactive proteasomal probe, is most suitable to report proteasomal activity in muscle cells using cell‐based bio‐imaging. We found that proteasomal activity is two‐fold and three‐fold enhanced in fused muscle cell culture compared with non‐fused cells. Moreover, we found that proteasomal activity can discriminate between muscles. Across muscles, a relative higher proteasomal activity was found in hybrid myofibers whereas fast‐twitch myofibers displayed lower activity.ConclusionsOur study demonstrates that proteasomal activity differ between muscles and between myofiber types. We suggest that ABPs can be used to report disease progression and treatment efficacy.

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Differential myofiber-type transduction preference of adeno-associated virus serotypes 6 and 9

Cited by 35 publications

References 34 publications

Mitochondrial glycerol 3‐phosphate dehydrogenase promotes skeletal muscle regeneration

Mitochondrial glycerol 3‐phosphate dehydrogenase promotes skeletal muscle regeneration

A compact unc45b‐promoter drives muscle‐specific expression in zebrafish and mouse

Proteasomal activity‐based probes mark protein homeostasis in muscles

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