Search Results - "Vafai, Scott"

Mitochondrial disorders as windows into an ancient organelle by Vafai, Scott B., Mootha, Vamsi K.

“…Much of our current knowledge about mitochondria has come from studying patients who have respiratory chain disorders. These disorders comprise a large…”
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Efficacy and Safety of an Investigational Single-Course CRISPR Base-Editing Therapy Targeting PCSK9 in Nonhuman Primate and Mouse Models by Lee, Richard G., Mazzola, Anne Marie, Braun, Maurine C., Platt, Colin, Vafai, Scott B., Kathiresan, Sekar, Rohde, Ellen, Bellinger, Andrew M., Khera, Amit V.

“…VERVE-101 is an investigational in vivo CRISPR base-editing medicine designed to alter a single DNA base in the gene, permanently turn off hepatic protein…”
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Mitochondrial dysfunction remodels one-carbon metabolism in human cells by Bao, Xiaoyan Robert, Ong, Shao-En, Goldberger, Olga, Peng, Jun, Sharma, Rohit, Thompson, Dawn A, Vafai, Scott B, Cox, Andrew G, Marutani, Eizo, Ichinose, Fumito, Goessling, Wolfram, Regev, Aviv, Carr, Steven A, Clish, Clary B, Mootha, Vamsi K

“…Mitochondrial dysfunction is associated with a spectrum of human disorders, ranging from rare, inborn errors of metabolism to common, age-associated diseases…”
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A Compendium of Genetic Modifiers of Mitochondrial Dysfunction Reveals Intra-organelle Buffering by To, Tsz-Leung, Cuadros, Alejandro M., Shah, Hardik, Hung, Wendy H.W., Li, Yang, Kim, Sharon H., Rubin, Daniel H.F., Boe, Ryan H., Rath, Sneha, Eaton, John K., Piccioni, Federica, Goodale, Amy, Kalani, Zohra, Doench, John G., Root, David E., Schreiber, Stuart L., Vafai, Scott B., Mootha, Vamsi K.

“…Mitochondrial dysfunction is associated with a spectrum of human conditions, ranging from rare, inborn errors of metabolism to the aging process. To identify…”
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Natural Product Screening Reveals Naphthoquinone Complex I Bypass Factors by Vafai, Scott B, Mevers, Emily, Higgins, Kathleen W, Fomina, Yevgenia, Zhang, Jianming, Mandinova, Anna, Newman, David, Shaw, Stanley Y, Clardy, Jon, Mootha, Vamsi K

“…Deficiency of mitochondrial complex I is encountered in both rare and common diseases, but we have limited therapeutic options to treat this lesion to the…”
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PGC1α Promotes Tumor Growth by Inducing Gene Expression Programs Supporting Lipogenesis by BHALLA, Kavita, HWANG, Bor Jang, GIRNUN, Geoffrey D, DEWI, Ruby E, OU, Lihui, TWADDEL, William, FANG, Hong-Bin, VAFAI, Scott B, VAZQUEZ, Francesca, PUIGSERVER, Pere, BOROS, Laszlo

“…Despite the role of aerobic glycolysis in cancer, recent studies highlight the importance of the mitochondria and biosynthetic pathways as well. PPARγ…”
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Effects of sodium benzoate, a widely used food preservative, on glucose homeostasis and metabolic profiles in humans by Lennerz, Belinda S., Vafai, Scott B., Delaney, Nigel F., Clish, Clary B., Deik, Amy A., Pierce, Kerry A., Ludwig, David S., Mootha, Vamsi K.

“…Sodium benzoate is a widely used preservative found in many foods and soft drinks. It is metabolized within mitochondria to produce hippurate, which is then…”
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A Mitochondrial Protein Compendium Elucidates Complex I Disease Biology by Pagliarini, David J., Calvo, Sarah E., Chang, Betty, Sheth, Sunil A., Vafai, Scott B., Ong, Shao-En, Walford, Geoffrey A., Sugiana, Canny, Boneh, Avihu, Chen, William K., Hill, David E., Vidal, Marc, Evans, James G., Thorburn, David R., Carr, Steven A., Mootha, Vamsi K.

“…Mitochondria are complex organelles whose dysfunction underlies a broad spectrum of human diseases. Identifying all of the proteins resident in this organelle…”
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Sympathetic Denervation of the Common Hepatic Artery Lessens Glucose Intolerance in the Fat- and Fructose-Fed Dog by Kraft, Guillaume, Vrba, Anthony, Scott, Melanie, Allen, Eric, Edgerton, Dale S, Williams, Phil E, Vafai, Scott B, Azamian, Bobak R, Cherrington, Alan D

“…This study assessed the effectiveness of surgical sympathetic denervation of the common hepatic artery (CHADN) in improving glucose tolerance. CHADN eliminated…”
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A Common Pathway for a Rare Disease? by Vafai, Scott B., Mootha, Vamsi K.

“…A rare childhood disorder caused by mitochondrial dysfunction is treated by the drug rapamycin in a mouse model of the disease. [Also see Report by Johnson et…”
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Regression of Drug-Resistant Lung Cancer by the Combination of Rosiglitazone and Carboplatin by GIRNUN, Geoffrey D, LIANG CHEN, NASERI, Elnaz, BUCKLEY, Stephanie, DANAN LI, FORCE, Jeremy, MCNAMARA, Kate, DEMETRI, George, SPIEGELMAN, Bruce M, WONG, Kwok-Kin, SILVAGGI, Jessica, DRAPKIN, Ronny, CHIRIEAC, Lucian R, PADERA, Robert F, UPADHYAY, Rabi, VAFAI, Scott B, WEISSLEDER, Ralph, MAHMOOD, Umar

“…Purpose: Current therapy for lung cancer involves multimodality therapies. However, many patients are either refractory to therapy or develop drug resistance…”
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Atypical case of Wolfram syndrome revealed through targeted exome sequencing in a patient with suspected mitochondrial disease by Lieber, Daniel S, Vafai, Scott B, Horton, Laura C, Slate, Nancy G, Liu, Shangtao, Borowsky, Mark L, Calvo, Sarah E, Schmahmann, Jeremy D, Mootha, Vamsi K

“…Mitochondrial diseases comprise a diverse set of clinical disorders that affect multiple organ systems with varying severity and age of onset. Due to their…”
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Protein phosphatase 2A methylation: a link between elevated plasma homocysteine and Alzheimer’s Disease by Vafai, Scott B, Stock, Jeffry B

“…Tau hyperphosphorylation is a central event in the development of Alzheimer’s Disease (AD). Protein phosphatase 2A (PP2A) heterotrimer formation is necessary…”
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Abstract 4139206: Design of Heart-2: a phase 1b clinical trial of VERVE-102, an in vivo base editing medicine delivered by a GalNAc-LNP and targeting PCSK9 to durably lower LDL cholesterol by Vafai, Scott, Karsten, Verena, Jensen, Chelsey, Falzone, Richard, Lister, Troy, Stolz, Leslie, Khera, Amit, Kathiresan, Sekar, Bellinger, Andrew, Fiedorek, Fred

“…Abstract only Introduction: Maintaining LDL-C at goal levels is critical in populations at high risk for cardiovascular events, including people with…”
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Abstract 11274: VERVE-101—An Investigational Single-Course Gene Editing Medicine Targeting PCSK9—Durably and Potently Lowers PCSK9 and LDL-C Concentrations in Non-Human Primates by Lee, Richard, Mazzola, Anne M, Braun, Maurine, Platt, Colin, Vafai, Scott, Kathiresan, Sekar, rohde, ellen, Bellinger, Andrew M, Khera, Amit V

“…IntroductionVERVE-101 is an in vivo CRISPR base editing therapy designed to alter a single DNA base in the PCSK9 gene, permanently inactivate hepatic…”
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Synergy between PPARγ Ligands and Platinum-Based Drugs in Cancer by Girnun, Geoffrey D., Naseri, Elnaz, Vafai, Scott B., Qu, Lishu, Szwaya, Jeffrey D., Bronson, Roderick, Alberta, John A., Spiegelman, Bruce M.

“…PPARγ is a member of the nuclear receptor family for which agonist ligands have antigrowth effects. However, clinical studies using PPARγ ligands as a…”
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Carboxyl methylation regulates phosphoprotein phosphatase 2A by controlling the association of regulatory B subunits by Tolstykh, Tatiana, Lee, Jookyung, Vafai, Scott, Stock, Jeffry B.

“…Phosphoprotein phosphatase 2A (PP2A) is a major phosphoserine/threonine protein phosphatase in all eukaryotes. It has been isolated as a heterotrimeric…”
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Medicine. A common pathway for a rare disease? by Vafai, Scott B, Mootha, Vamsi K

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Synergy between PPARgamma ligands and platinum-based drugs in cancer by Girnun, Geoffrey D, Naseri, Elnaz, Vafai, Scott B, Qu, Lishu, Szwaya, Jeffrey D, Bronson, Roderick, Alberta, John A, Spiegelman, Bruce M

“…PPARgamma is a member of the nuclear receptor family for which agonist ligands have antigrowth effects. However, clinical studies using PPARgamma ligands as a…”
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