Rescue of Mutant CFTR Trafficking Defect by the Investigational Compound MCG1516A Article Swipe
Miquéias Lopes‐Pacheco
,
Mafalda Bacalhau
,
Sofia S. Ramalho
,
Iris A. L. Silva
,
Filipa C. Ferreira
,
Graeme W. Carlile
,
David Y. Thomas
,
Carlos M. Farinha
,
John W. Hanrahan
,
Margarida D. Amaral
·
YOU?
·
· 2022
· Open Access
·
· DOI: https://doi.org/10.3390/cells11010136
YOU?
·
· 2022
· Open Access
·
· DOI: https://doi.org/10.3390/cells11010136
Although some therapeutic progress has been achieved in developing small molecules that correct F508del-CFTR defects, the mechanism of action (MoA) of these compounds remain poorly elucidated. Here, we investigated the effects and MoA of MCG1516A, a newly developed F508del-CFTR corrector. MCG1516A effects on wild-type (WT) and F508del-CFTR were assessed by immunofluorescence microscopy, and biochemical and functional assays both in cell lines and in intestinal organoids. To shed light on the MoA of MCG1516A, we evaluated its additivity to the FDA-approved corrector VX-661, low temperature, genetic revertants of F508del-CFTR (G550E, R1070W, and 4RK), and the traffic-null variant DD/AA. Finally, we explored the ability of MCG1516A to rescue trafficking and function of other CF-causing mutations. We found that MCG1516A rescues F508del-CFTR with additive effects to VX-661. A similar behavior was observed for WT-CFTR. Under low temperature incubation, F508del-CFTR demonstrated an additivity in processing and function with VX-661, but not with MCG1516A. In contrast, both compounds promoted additional effects to low temperature to WT-CFTR. MCG1516A demonstrated additivity to genetic revertant R1070W, while VX-661 was additive to G550E and 4RK. Nevertheless, none of these compounds rescued DD/AA trafficking. Both MCG1516A and VX-661 rescued CFTR processing of L206W- and R334W-CFTR with greater effects when these compounds were combined. In summary, the absence of additivity of MCG1516A to genetic revertant G550E suggests a putative binding site for this compound on NBD1:NBD2 interface. Therefore, a combination of MCG1516A with compounds able to rescue DD/AA traffic, or mimicking the actions of revertant R1070W (e.g., VX-661), could enhance correction of F508del-CFTR defects.
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Metadata
- Type
- article
- Language
- en
- Landing Page
- https://doi.org/10.3390/cells11010136
- https://www.mdpi.com/2073-4409/11/1/136/pdf?version=1641029508
- OA Status
- gold
- Cited By
- 15
- References
- 45
- Related Works
- 10
- OpenAlex ID
- https://openalex.org/W4206255712
All OpenAlex metadata
Raw OpenAlex JSON
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https://openalex.org/W4206255712Canonical identifier for this work in OpenAlex
- DOI
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https://doi.org/10.3390/cells11010136Digital Object Identifier
- Title
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Rescue of Mutant CFTR Trafficking Defect by the Investigational Compound MCG1516AWork title
- Type
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articleOpenAlex work type
- Language
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enPrimary language
- Publication year
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2022Year of publication
- Publication date
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2022-01-01Full publication date if available
- Authors
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Miquéias Lopes‐Pacheco, Mafalda Bacalhau, Sofia S. Ramalho, Iris A. L. Silva, Filipa C. Ferreira, Graeme W. Carlile, David Y. Thomas, Carlos M. Farinha, John W. Hanrahan, Margarida D. AmaralList of authors in order
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https://doi.org/10.3390/cells11010136Publisher landing page
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https://www.mdpi.com/2073-4409/11/1/136/pdf?version=1641029508Direct link to full text PDF
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YesWhether a free full text is available
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goldOpen access status per OpenAlex
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https://www.mdpi.com/2073-4409/11/1/136/pdf?version=1641029508Direct OA link when available
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Mutant, Chemistry, Mutation, Wild type, Molecular biology, Cell biology, Pharmacology, Biology, Biochemistry, GeneTop concepts (fields/topics) attached by OpenAlex
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15Total citation count in OpenAlex
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2025: 4, 2024: 4, 2023: 2, 2022: 5Per-year citation counts (last 5 years)
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45Number of works referenced by this work
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-
10Other works algorithmically related by OpenAlex
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