Direct Expression of CPT1a Enables a High Throughput Platform for the Discovery of CPT1a Modulators

Carnitine palmitoyltransferase 1 (CPT1), which catalyzes the rate-limiting step of fatty acid oxidation, has been implicated in therapeutic approaches to several human diseases characterized by aberrant lipid metabolism. The isoform-specific quantification of CPT1 activity is essential in the charac...

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Main Authors: Jason Chen, Tuyen Tran, Anthony Wong, Luofei Wang, Pranavi Annaluru, Vibha Sreekanth, Samika Murthy, Laasya Munjeti, Tanya Park, Utkarsh Bhat, Glynnis Leong, Yumeng Li, Simeng Chen, Natalie Kong, Rushika Raval, Yining Xie, Shreya Somani, Aditi Manohar Bhambhani, Zoey Zhu, Landen Chu, Kimai Dosch, Edward Njoo, Zhan Chen
Format: Article
Language:English
Published: MDPI AG 2025-05-01
Series:Applied Biosciences
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Online Access:https://www.mdpi.com/2813-0464/4/2/25
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author Jason Chen
Tuyen Tran
Anthony Wong
Luofei Wang
Pranavi Annaluru
Vibha Sreekanth
Samika Murthy
Laasya Munjeti
Tanya Park
Utkarsh Bhat
Glynnis Leong
Yumeng Li
Simeng Chen
Natalie Kong
Rushika Raval
Yining Xie
Shreya Somani
Aditi Manohar Bhambhani
Zoey Zhu
Landen Chu
Kimai Dosch
Edward Njoo
Zhan Chen
author_facet Jason Chen
Tuyen Tran
Anthony Wong
Luofei Wang
Pranavi Annaluru
Vibha Sreekanth
Samika Murthy
Laasya Munjeti
Tanya Park
Utkarsh Bhat
Glynnis Leong
Yumeng Li
Simeng Chen
Natalie Kong
Rushika Raval
Yining Xie
Shreya Somani
Aditi Manohar Bhambhani
Zoey Zhu
Landen Chu
Kimai Dosch
Edward Njoo
Zhan Chen
author_sort Jason Chen
collection DOAJ
description Carnitine palmitoyltransferase 1 (CPT1), which catalyzes the rate-limiting step of fatty acid oxidation, has been implicated in therapeutic approaches to several human diseases characterized by aberrant lipid metabolism. The isoform-specific quantification of CPT1 activity is essential in the characterization of small molecule inhibitors of CPT1, but several existing means to quantify enzymatic activity, including the use of radioisotope-labeled carnitine, are not amenable to scalable, high throughput screening. Here, we demonstrate that mitochondrial extracts from Expi293 cells transfected with a CPT1a plasmid are a reliable and robust source of catalytically active human CPT1. Moreover, with a source of catalytically active enzyme in hand, we modified a previously reported colorimetric method of coenzyme A (CoA) easily scalable to a 96-well format for the screening of CPT1a inhibitors. This assay platform was validated by two previously reported inhibitors of CPT1a: <i>R-</i>etomoxir and perhexiline. To further demonstrate the applicability of this method in small molecule screening, we prepared and screened a library of 87 known small molecule APIs, validating the inhibitory effect of chlorpromazine on CPT1.
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spelling doaj-art-bc930a9a762c4ec89b45e4347c35f3e72025-08-20T02:24:22ZengMDPI AGApplied Biosciences2813-04642025-05-01422510.3390/applbiosci4020025Direct Expression of CPT1a Enables a High Throughput Platform for the Discovery of CPT1a ModulatorsJason Chen0Tuyen Tran1Anthony Wong2Luofei Wang3Pranavi Annaluru4Vibha Sreekanth5Samika Murthy6Laasya Munjeti7Tanya Park8Utkarsh Bhat9Glynnis Leong10Yumeng Li11Simeng Chen12Natalie Kong13Rushika Raval14Yining Xie15Shreya Somani16Aditi Manohar Bhambhani17Zoey Zhu18Landen Chu19Kimai Dosch20Edward Njoo21Zhan Chen22Department of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Biological, Human and Life Sciences, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Biological, Human and Life Sciences, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Biological, Human and Life Sciences, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Biological, Human and Life Sciences, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Biological, Human and Life Sciences, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Biological, Human and Life Sciences, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Biological, Human and Life Sciences, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Biological, Human and Life Sciences, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Biological, Human and Life Sciences, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USADepartment of Chemistry, Biochemistry and Physics, Aspiring Scholars Directed Research Program, Fremont, CA 94539, USACarnitine palmitoyltransferase 1 (CPT1), which catalyzes the rate-limiting step of fatty acid oxidation, has been implicated in therapeutic approaches to several human diseases characterized by aberrant lipid metabolism. The isoform-specific quantification of CPT1 activity is essential in the characterization of small molecule inhibitors of CPT1, but several existing means to quantify enzymatic activity, including the use of radioisotope-labeled carnitine, are not amenable to scalable, high throughput screening. Here, we demonstrate that mitochondrial extracts from Expi293 cells transfected with a CPT1a plasmid are a reliable and robust source of catalytically active human CPT1. Moreover, with a source of catalytically active enzyme in hand, we modified a previously reported colorimetric method of coenzyme A (CoA) easily scalable to a 96-well format for the screening of CPT1a inhibitors. This assay platform was validated by two previously reported inhibitors of CPT1a: <i>R-</i>etomoxir and perhexiline. To further demonstrate the applicability of this method in small molecule screening, we prepared and screened a library of 87 known small molecule APIs, validating the inhibitory effect of chlorpromazine on CPT1.https://www.mdpi.com/2813-0464/4/2/25carnitine palmitoyltransferase activity assayCPT1 inhibitorCPT1 modulatorsfatty acid oxidation
spellingShingle Jason Chen
Tuyen Tran
Anthony Wong
Luofei Wang
Pranavi Annaluru
Vibha Sreekanth
Samika Murthy
Laasya Munjeti
Tanya Park
Utkarsh Bhat
Glynnis Leong
Yumeng Li
Simeng Chen
Natalie Kong
Rushika Raval
Yining Xie
Shreya Somani
Aditi Manohar Bhambhani
Zoey Zhu
Landen Chu
Kimai Dosch
Edward Njoo
Zhan Chen
Direct Expression of CPT1a Enables a High Throughput Platform for the Discovery of CPT1a Modulators
Applied Biosciences
carnitine palmitoyltransferase activity assay
CPT1 inhibitor
CPT1 modulators
fatty acid oxidation
title Direct Expression of CPT1a Enables a High Throughput Platform for the Discovery of CPT1a Modulators
title_full Direct Expression of CPT1a Enables a High Throughput Platform for the Discovery of CPT1a Modulators
title_fullStr Direct Expression of CPT1a Enables a High Throughput Platform for the Discovery of CPT1a Modulators
title_full_unstemmed Direct Expression of CPT1a Enables a High Throughput Platform for the Discovery of CPT1a Modulators
title_short Direct Expression of CPT1a Enables a High Throughput Platform for the Discovery of CPT1a Modulators
title_sort direct expression of cpt1a enables a high throughput platform for the discovery of cpt1a modulators
topic carnitine palmitoyltransferase activity assay
CPT1 inhibitor
CPT1 modulators
fatty acid oxidation
url https://www.mdpi.com/2813-0464/4/2/25
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