#METABOLOMICS WORKBENCH bendovi_20230131_091306 DATATRACK_ID:3717 STUDY_ID:ST002458 ANALYSIS_ID:AN004010 PROJECT_ID:PR001585 VERSION 1 CREATED_ON January 31, 2023, 11:25 am #PROJECT PR:PROJECT_TITLE Mouse kidney metabolomic studies PR:PROJECT_TYPE MS quantitative analysis PR:PROJECT_SUMMARY Chronic kidney disease secondary to cystic kidney disease is a leading cause of PR:PROJECT_SUMMARY mortality in patients with tuberous sclerosis complex (TSC) disease. The PR:PROJECT_SUMMARY mechanisms underlying TSC cystic kidney disease are obscure, with no PR:PROJECT_SUMMARY interventions available to prevent cyst formation. Here, we reveal for the first PR:PROJECT_SUMMARY time the misregulated metabolic pathways in TSC kidneys and their relevance to PR:PROJECT_SUMMARY TSC-associated cytogenesis. To this end, we have analyzed the metabolic profile PR:PROJECT_SUMMARY of the whole kidney as well as sorted proximal tubule cell (PTCs) extracts. The PR:PROJECT_SUMMARY metabolomics data show that Tsc1 deletion in nephron progenitor cells changes PR:PROJECT_SUMMARY the arginine biosynthesis pathway as well as a substantial number of metabolic PR:PROJECT_SUMMARY pathways. These changes were associated with overexpression of argininosuccinate PR:PROJECT_SUMMARY synthetase 1 (ASS1), a rate-limiting enzyme in the arginine biosynthetic pathway PR:PROJECT_SUMMARY in TSC KO mice and human kidneys. The rise in ASS1 expression is dependent on PR:PROJECT_SUMMARY mTORC1 activity. Arginine depletion in vitro and in vivo prevented the rise in PR:PROJECT_SUMMARY mTORC1 activity and cell cycle progression and averted the overexpression of PR:PROJECT_SUMMARY previously described cytogenetic signaling proteins, including c-Myc and P65. PR:PROJECT_SUMMARY Accordingly, an arginine-depleted diet substantially reduced the TSC cystic PR:PROJECT_SUMMARY load, indicating the potential therapeutic effects of arginine deprivation as a PR:PROJECT_SUMMARY treatment of TSC-associated kidney disease. PR:INSTITUTE Hadassah Medical Center PR:DEPARTMENT Pediatric Nephrology PR:LABORATORY Wohl Institute for Translational Medicine PR:LAST_NAME Ben-Dov PR:FIRST_NAME Iddo PR:ADDRESS Ein Karem Campus, Jerusalem, NA, 9211001, Israel PR:EMAIL iddo@hadassah.org.il PR:PHONE +97226776881 PR:FUNDING_SOURCE This work was supported by grants from the Israel Science Foundation (MN 2030/21 PR:FUNDING_SOURCE and OV 2358/18), the TSC alliance research grant (OV), the research authority of PR:FUNDING_SOURCE Hadassah Hebrew University Medical Center (OV). OV and MN are Wohl’s PR:FUNDING_SOURCE Translation Research Institute research associates at Hadassah-Hebrew University PR:FUNDING_SOURCE Medical Center PR:CONTRIBUTORS Athar Amleh, Lana Watad, Ifat Abramovich, Bella Agranovich, Eyal Gottlieb, Iddo PR:CONTRIBUTORS Z. Ben-Dov, Morris Nechama and Oded Volovelsky #STUDY ST:STUDY_TITLE Mouse kidney metabolomics (Proximal tubular cells) ST:STUDY_SUMMARY Here, we reveal for the first time the misregulated metabolic pathways in TSC ST:STUDY_SUMMARY kidneys and their relevance to TSC-associated cytogenesis. To this end, we have ST:STUDY_SUMMARY analyzed the metabolic profile of the whole kidney as well as sorted proximal ST:STUDY_SUMMARY tubule cell (PTCs) extracts. The metabolomics data show that Tsc1 deletion in ST:STUDY_SUMMARY nephron progenitor cells changes the arginine biosynthesis pathway as well as a ST:STUDY_SUMMARY substantial number of metabolic pathways. ST:INSTITUTE Hadassah Medical Center ST:LAST_NAME Ben-Dov ST:FIRST_NAME Iddo ST:ADDRESS Hadassah Medical Center, Jerusalem, Israel 91120 ST:EMAIL iddo@hadassah.org.il ST:NUM_GROUPS 3 ST:TOTAL_SUBJECTS 26 ST:STUDY_TYPE MS quantitative analysis ST:PHONE +97226776881 #SUBJECT SU:SUBJECT_TYPE Mammal SU:SUBJECT_SPECIES Mus musculus SU:TAXONOMY_ID 10090 SU:GENDER Male and female #SUBJECT_SAMPLE_FACTORS: SUBJECT(optional)[tab]SAMPLE[tab]FACTORS(NAME:VALUE pairs separated by |)[tab]Raw file names and additional sample data SUBJECT_SAMPLE_FACTORS 1 01_WT_1 Genotype:Wild-type | Treatment:none RAW_FILE_NAME=01_WT_1.raw SUBJECT_SAMPLE_FACTORS 2 02_WT_2 Genotype:Wild-type | Treatment:none RAW_FILE_NAME=02_WT_2.raw SUBJECT_SAMPLE_FACTORS 3 03_WT_3 Genotype:Wild-type | Treatment:none RAW_FILE_NAME=03_WT_3.raw SUBJECT_SAMPLE_FACTORS 4 04_KO_1 Genotype:Tsc1-knockout | Treatment:none RAW_FILE_NAME=04_KO_1.raw SUBJECT_SAMPLE_FACTORS 5 05_KO_2 Genotype:Tsc1-knockout | Treatment:none RAW_FILE_NAME=05_KO_2.raw SUBJECT_SAMPLE_FACTORS 6 06_KO_3 Genotype:Tsc1-knockout | Treatment:none RAW_FILE_NAME=06_KO_3.raw SUBJECT_SAMPLE_FACTORS 7 07_KO_Rapha1 Genotype:Tsc1-knockout | Treatment:rapamycin RAW_FILE_NAME=07_KO_Rapha1.raw SUBJECT_SAMPLE_FACTORS 8 08_KO_Rapha2 Genotype:Tsc1-knockout | Treatment:rapamycin RAW_FILE_NAME=08_KO_Rapha2.raw SUBJECT_SAMPLE_FACTORS 9 09_KO_Rapha3 Genotype:Tsc1-knockout | Treatment:rapamycin RAW_FILE_NAME=09_KO_Rapha3.raw #COLLECTION CO:COLLECTION_SUMMARY PTCs were isolated as previously described(PMID: 32484794). Briefly, kidneys CO:COLLECTION_SUMMARY were excised in ice-cold HBSS buffer. The kidneys were sliced and chopped into CO:COLLECTION_SUMMARY pieces (~0.5–1 mm) on ice using a surgical scalpel. The chopped kidneys were CO:COLLECTION_SUMMARY transferred into an HBSS solution containing 1 μg/μL collagenase/dispase CO:COLLECTION_SUMMARY (Sigma Aldrich, Cat. # 10269638001) and incubated for 25 minutes at 370 C. The CO:COLLECTION_SUMMARY cells were filtered through a 40-μm nylon cell strainer (Corning, Cat. # CO:COLLECTION_SUMMARY 431750) and washed twice with cold HBSS. For PTC isolation, the cells were CO:COLLECTION_SUMMARY stained with PE-conjugated antiCD133/prominin-1 antibody (Invitrogen, Cat. # CO:COLLECTION_SUMMARY 12-1331-82) according to the manufacturer's instructions. PE+ cells were CO:COLLECTION_SUMMARY isolated by Aria III flow cytometry-based cell sorting. CO:SAMPLE_TYPE Proximal tubular cells #TREATMENT TR:TREATMENT_SUMMARY A mixture of six labeled internal standards was added to the extraction solution TR:TREATMENT_SUMMARY for quality control (13C6-Glucose, 13C5-Glutamine, 13C5-Glutamate, 13C1-Alanine, TR:TREATMENT_SUMMARY 13C3-Pyruvate and 13C3-Lactate). The exact volume at each tube was adjusted TR:TREATMENT_SUMMARY according to tissue weight (average volume of 200 µl). The samples were TR:TREATMENT_SUMMARY homogenized using Precellys 24 tissue homogenizer (Bertin Technologies) TR:TREATMENT_SUMMARY precooled to 4°C (3 cycles × 30 s at 6000 rpm, with a 30 s gap TR:TREATMENT_SUMMARY between each cycle) and later centrifuged at 18,000 g for 15 min at 4°C. The TR:TREATMENT_SUMMARY supernatants were transferred into HPLC glass vials and kept at −80°C prior TR:TREATMENT_SUMMARY to LC-MS analysis. #SAMPLEPREP SP:SAMPLEPREP_SUMMARY The samples were homogenized using Precellys 24 tissue homogenizer (Bertin SP:SAMPLEPREP_SUMMARY Technologies) precooled to 4°C (3 cycles × 30 s at 6000 rpm, with a SP:SAMPLEPREP_SUMMARY 30 s gap between each cycle) and later centrifuged at 18,000 g for 15 min at SP:SAMPLEPREP_SUMMARY 4°C. The supernatants were transferred into HPLC glass vials and kept at SP:SAMPLEPREP_SUMMARY −80°C prior to LC-MS analysis. #CHROMATOGRAPHY CH:CHROMATOGRAPHY_SUMMARY UPLC setup consisted a ZIC-pHILIC column (SeQuant; 150 mm × 2.1 mm, CH:CHROMATOGRAPHY_SUMMARY 5 μm; Merck). 5 µl of cells or kidney extracts were injected using an CH:CHROMATOGRAPHY_SUMMARY autosampler. Compounds were separated using a 15 minutes gradient, starting at CH:CHROMATOGRAPHY_SUMMARY 20% aqueous (20 mM ammonium carbonate adjusted to pH 9.2 with 0.1% of 25% CH:CHROMATOGRAPHY_SUMMARY ammonium hydroxide) and 80% organic (acetonitrile), terminated with 20% CH:CHROMATOGRAPHY_SUMMARY acetonitrile. Flow rate and column temperature were kept at 0.2 ml/min and CH:CHROMATOGRAPHY_SUMMARY 45°C, respectively for a total run time of 27 min CH:CHROMATOGRAPHY_TYPE HILIC CH:INSTRUMENT_NAME Thermo Dionex Ultimate 3000 CH:COLUMN_NAME Merck SeQuant ZIC-pHILIC (150 x 2.1mm,5um) CH:SOLVENT_A 20% water/80% acetonitrile; 20 mM ammonium carbonate; 0.1% of 25% ammonium CH:SOLVENT_A hydroxide (adjusted to pH 9.2) CH:SOLVENT_B 80%water/20% acetonitrile CH:FLOW_GRADIENT 15 min terminated with 20% acetonitrile CH:FLOW_RATE 0.2 ml/min CH:COLUMN_TEMPERATURE 45 #ANALYSIS AN:ANALYSIS_TYPE MS AN:LABORATORY_NAME Technion Mass Spect #MS MS:INSTRUMENT_NAME Thermo Q Exactive Orbitrap MS:INSTRUMENT_TYPE Orbitrap MS:MS_TYPE Other MS:ION_MODE UNSPECIFIED MS:MS_COMMENTS Briefly, Dionex Ultimate ultra-high-performance liquid chromatography (UPLC) MS:MS_COMMENTS system coupled to Orbitrap Q-Exactive mass spectrometer (Thermo Fisher MS:MS_COMMENTS Scientific) was used. The resolution was set to 35,000 at 200 mass/charge ratio MS:MS_COMMENTS (m/z) with electrospray ionization and polarity switching mode to enable both MS:MS_COMMENTS positive and negative ions across a mass range of 67–1000 m/z. #MS_METABOLITE_DATA MS_METABOLITE_DATA:UNITS concentration MS_METABOLITE_DATA_START Samples 04_KO_1 05_KO_2 06_KO_3 07_KO_Rapha1 08_KO_Rapha2 09_KO_Rapha3 01_WT_1 02_WT_2 03_WT_3 Factors Genotype:Tsc1-knockout | Treatment:none Genotype:Tsc1-knockout | Treatment:none Genotype:Tsc1-knockout | Treatment:none Genotype:Tsc1-knockout | Treatment:rapamycin Genotype:Tsc1-knockout | Treatment:rapamycin Genotype:Tsc1-knockout | Treatment:rapamycin Genotype:Wild-type | Treatment:none Genotype:Wild-type | Treatment:none Genotype:Wild-type | Treatment:none pyroglutamic acid 2120000 2440000 2570000 909000 1020000 647000 4750000 1640000 813000 acetyl-aspartate (N) 539000 464000 487000 118000 117000 43800 61400 173000 0 acetyl-carnitine 9830000 10500000 9320000 4560000 4720000 3290000 5270000 4460000 1590000 Adenine 831000 592000 750000 983000 876000 706000 903000 438000 250000 Adenosine 288000 288000 279000 1020000 521000 357000 870000 387000 67900 aKG 1540000 278000 442000 1100000 192000 107000 235000 0 0 Alanine 3430000 3750000 3570000 3180000 3410000 1830000 1610000 1950000 742000 aminoadipate 129000 140000 106000 247000 215000 128000 131000 98900 82300 Arachidonic acid 0 258000 0 792000 839000 0 281000 510000 0 Arginine 812000 829000 800000 635000 478000 667000 2960000 674000 259000 Asparagine 838000 1020000 802000 618000 633000 310000 342000 1080000 21000 Aspartate 6730000 6190000 5930000 5180000 5200000 3270000 4050000 3380000 1310000 betaine 19300000 16300000 16400000 9340000 7070000 6390000 14000000 16800000 1990000 Butyric acid 1400000 932000 797000 950000 712000 996000 1100000 731000 407000 Butyryl-carnitine 184000 209000 161000 121000 164000 44200 104000 78200 40900 carnitine 17500000 16400000 14000000 12700000 13100000 8170000 8000000 7750000 2420000 Citrulline 823000 834000 876000 337000 366000 257000 256000 132000 0 Creatinine 335000 352000 254000 404000 454000 173000 95500 137000 7160 Decanoic acid 3020000 4380000 1340000 1120000 1160000 672000 1160000 1620000 2130000 Dodecanoic acid/Lauric acid 7500000 8990000 1330000 943000 604000 1080000 1690000 3790000 5050000 Eicosapentaenoic acid 80800 1450000 0 1500000 1600000 0 989000 1840000 307000 Ethanolamine phosphate 3700000 3520000 3090000 1750000 1940000 1790000 1340000 1190000 0 Glucose 3.14E+08 3.07E+08 2.57E+08 6.88E+08 6.81E+08 4.56E+08 4.61E+08 4.66E+08 1.76E+08 Glutamate 14400000 15500000 13300000 12700000 12400000 7170000 9700000 7420000 2500000 Glutamine 3220000 3140000 3020000 3480000 3240000 2180000 1980000 2090000 469000 Glycine 2900000 3160000 3480000 1230000 1430000 640000 18700000 19400000 5810000 hypoxanthine 202000 161000 189000 110000 110000 30700 152000 148000 7500 Isocapric Acid 1300000 1340000 1010000 1040000 1020000 991000 1100000 1020000 682000 Lactate 13000000 14000000 2610000 4650000 3040000 3040000 5310000 4750000 9180000 Leucine 6860000 6840000 6740000 5480000 5510000 3740000 4960000 5220000 1790000 Linoleic acid 1530000 1140000 1010000 1130000 1080000 3260000 1840000 1640000 3150000 Lysine 434000 585000 311000 482000 567000 211000 381000 295000 201000 Malate 3000000 3100000 2820000 3830000 916000 811000 3060000 2160000 341000 Methionine 1580000 1780000 1340000 1140000 1370000 578000 1100000 884000 136000 Myristic acid 7690000 9670000 3590000 2790000 1900000 2510000 3640000 5710000 5880000 NAD+ 191000 295000 120000 293000 285000 66200 72300 66700 0 nicotinamide 1330000 1440000 1200000 1160000 1180000 1160000 1400000 1280000 691000 Octanoic acid 2960000 4090000 1620000 1890000 1580000 1050000 1750000 2260000 1210000 Oleic acid 6890000 5280000 3820000 5650000 3430000 18500000 7770000 7740000 17700000 Palmitic acid 54800000 64900000 76100000 61200000 36400000 32100000 54900000 51800000 27100000 Palmitoleic acid 6320000 7890000 2550000 2270000 1580000 3160000 4110000 7130000 8370000 PEP 482000 292000 108000 72000 133000 218000 299000 83000 0 Phenylalanine 2720000 2720000 2500000 2160000 2040000 1610000 2240000 1460000 504000 Phosphocreatine 201000 344000 199000 10500 126000 222000 12300 30600 0 Proline 9650000 9400000 9570000 8340000 8230000 9220000 18100000 5980000 2000000 Propionyl-carnitine 1190000 1150000 957000 885000 917000 611000 566000 664000 388000 Pyruvate 1300000 948000 427000 1020000 519000 864000 1540000 551000 583000 Ribose 8570000 8400000 6750000 18900000 19200000 12800000 12700000 13100000 4980000 S-Adenosyl-L-methionine 162000 180000 132000 79500 332000 85200 11600 29000 0 Serine 622000 709000 674000 420000 385000 181000 212000 170000 7050 Stearic acid 33600000 40500000 54500000 37200000 26500000 21200000 36400000 31700000 15700000 Succinic Acid 1830000 335000 827000 1070000 209000 1560000 259000 0 1100000 Taurine 9850000 9370000 8220000 5370000 5500000 3770000 3310000 3370000 1310000 Threonine 2800000 3350000 3100000 2190000 2700000 2150000 1730000 2140000 1290000 Tryptophan 488000 507000 368000 210000 221000 143000 215000 196000 0 Tyrosine 307000 399000 287000 165000 145000 158000 141000 128000 39100 UDP-GlcNAc 153000 177000 44300 103000 137000 56600 22700 55900 8930 MS_METABOLITE_DATA_END #METABOLITES METABOLITES_START metabolite_name pyroglutamic acid acetyl-aspartate (N) acetyl-carnitine Adenine Adenosine aKG Alanine aminoadipate Arachidonic acid Arginine Asparagine Aspartate betaine Butyric acid Butyryl-carnitine carnitine Citrulline Creatinine Decanoic acid Dodecanoic acid/Lauric acid Eicosapentaenoic acid Ethanolamine phosphate Glucose Glutamate Glutamine Glycine hypoxanthine Isocapric Acid Lactate Leucine Linoleic acid Lysine Malate Methionine Myristic acid NAD+ nicotinamide Octanoic acid Oleic acid Palmitic acid Palmitoleic acid PEP Phenylalanine Phosphocreatine Proline Propionyl-carnitine Pyruvate Ribose S-Adenosyl-L-methionine Serine Stearic acid Succinic Acid Taurine Threonine Tryptophan Tyrosine UDP-GlcNAc METABOLITES_END #END