Search Results - "Khoja, Suhail"

Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency by Khoja, Suhail, Liu, Xiao-Bo, Truong, Brian, Nitzahn, Matthew, Lambert, Jenna, Eliav, Adam, Nasser, Eram, Randolph, Emma, Burke, Kristine E., White, Rebecca, Zhu, Xuling, Martini, Paolo G.V., Nissim, Itzhak, Cederbaum, Stephen D., Lipshutz, Gerald S.

“…Arginase deficiency is associated with prominent neuromotor features, including spastic diplegia, clonus, and hyperreflexia; intellectual disability and…”
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Noncoding Centromeric RNA Expression Impairs Chromosome Stability in Human and Murine Stem Cells by Monaco, Zoia L., Khoja, Suhail, Moralli, Daniela, Chan, David Yiu Leung

“…We analyzed the effect of transcribed noncoding RNA centromeric satellites on chromosome segregation in normal human and murine stem and fibrosarcoma cells…”
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Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in mice by Angarita, Stephanie A.K., Truong, Brian, Khoja, Suhail, Nitzahn, Matthew, Rajbhandari, Abha K., Zhuravka, Irina, Duarte, Sergio, Lin, Michael G., Lam, Alex K., Cederbaum, Stephen D., Lipshutz, Gerald S.

“…The transplantation, engraftment, and expansion of primary hepatocytes have the potential to be an effective therapy for metabolic disorders of the liver…”
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Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities by Khoja, Suhail, Lambert, Jenna, Nitzahn, Matthew, Eliav, Adam, Zhang, YuChen, Tamboline, Mikayla, Le, Colleen T., Nasser, Eram, Li, Yunfeng, Patel, Puja, Zhuravka, Irina, Lueptow, Lindsay M., Tkachyova, Ilona, Xu, Shili, Nissim, Itzhak, Schulze, Andreas, Lipshutz, Gerald S.

“…Creatine deficiency disorders are inborn errors of creatine metabolism, an energy homeostasis molecule. One of these, guanidinoacetate N-methyltransferase…”
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Magnetic Resonance Imaging of Iron Oxide‐Labeled Human Embryonic Stem Cell‐Derived Cardiac Progenitors by Skelton, Rhys J.P., Khoja, Suhail, Almeida, Shone, Rapacchi, Stanislas, Han, Fei, Engel, James, Zhao, Peng, Hu, Peng, Stanley, Edouard G., Elefanty, Andrew G., Kwon, Murray, Elliott, David A., Ardehali, Reza

“…Given the limited regenerative capacity of the heart, cellular therapy with stem cell‐derived cardiac cells could be a potential treatment for patients with…”
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CD13 and ROR2 Permit Isolation of Highly Enriched Cardiac Mesoderm from Differentiating Human Embryonic Stem Cells by Skelton, Rhys J.P., Brady, Bevin, Khoja, Suhail, Sahoo, Debashis, Engel, James, Arasaratnam, Deevina, Saleh, Kholoud K., Abilez, Oscar J., Zhao, Peng, Stanley, Edouard G., Elefanty, Andrew G., Kwon, Murray, Elliott, David A., Ardehali, Reza

“…The generation of tissue-specific cell types from human embryonic stem cells (hESCs) is critical for the development of future stem cell-based regenerative…”
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Genetic analysis of HIV-1 subtypes in Nairobi, Kenya by Khoja, Suhail, Ojwang, Peter, Khan, Saeed, Okinda, Nancy, Harania, Reena, Ali, Syed

“…Genetic analysis of a viral infection helps in following its spread in a given population, in tracking the routes of infection and, where applicable, in…”
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Functional human artificial chromosomes are generated and stably maintained in human embryonic stem cells by Mandegar, Mohammad A., Moralli, Daniela, Khoja, Suhail, Cowley, Sally, Chan, David Y.L., Yusuf, Mohammed, Mukherjee, Sayandip, Blundell, Michael P., Volpi, Emanuela V., Thrasher, Adrian J., James, William, Monaco, Zoia L.

“…We present a novel and efficient non-integrating gene expression system in human embryonic stem cells (hESc) utilizing human artificial chromosomes (HAC),…”
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Evidence for a "Founder Effect" among HIV-infected injection drug users (IDUs) in Pakistan by Rai, Mohammad A, Nerurkar, Vivek R, Khoja, Suhail, Khan, Saeed, Yanagihara, Richard, Rehman, Arish, Kazmi, Shahana U, Ali, Syed H

“…We have previously reported a HIV-1 subtype A infection in a community of injection drug users (IDUs) in Karachi, Pakistan. We now show that this infection…”
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Split AAV-Mediated Gene Therapy Restores Ureagenesis in a Murine Model of Carbamoyl Phosphate Synthetase 1 Deficiency by Nitzahn, Matthew, Allegri, Gabriella, Khoja, Suhail, Truong, Brian, Makris, Georgios, Häberle, Johannes, Lipshutz, Gerald S.

“…The urea cycle enzyme carbamoyl phosphate synthetase 1 (CPS1) catalyzes the initial step of the urea cycle; bi-allelic mutations typically present with…”
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A constitutive knockout of murine carbamoyl phosphate synthetase 1 results in death with marked hyperglutaminemia and hyperammonemia by Khoja, Suhail, Nitzahn, Matthew, Truong, Brian, Lambert, Jenna, Willis, Brandon, Allegri, Gabriella, Rüfenacht, Véronique, Häberle, Johannes, Lipshutz, Gerald S.

“…The enzyme carbamoyl phosphate synthetase 1 (CPS1; EC 6.3.4.16) forms carbamoyl phosphate from bicarbonate, ammonia, and adenosine triphosphate (ATP) and is…”
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Conditional disruption of hepatic carbamoyl phosphate synthetase 1 in mice results in hyperammonemia without orotic aciduria and can be corrected by liver-directed gene therapy by Khoja, Suhail, Nitzahn, Matt, Hermann, Kip, Truong, Brian, Borzone, Roberta, Willis, Brandon, Rudd, Mitchell, Palmer, Donna J., Ng, Philip, Brunetti-Pierri, Nicola, Lipshutz, Gerald S.

“…Carbamoyl phosphate synthetase 1 (CPS1) is a urea cycle enzyme that forms carbamoyl phosphate from bicarbonate, ammonia and ATP. Bi-allelic mutations of the…”
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Abstract 461: Prospective Isolation of Heart Field Specific Cardiomyocytes from Differentiating Human Embryonic Stem Cells by Engel, James L, Pezhouman, Arash, Nguyen, Ngoc B, Zhao, Peng, Khoja, Suhail, Skelton, Rhys J, Sahoo, Debashis, Elliott, David A, Ardehali, Reza

“…Abstract only Cardiovascular disease (CVD) is the leading cause of death worldwide. Human embryonic stem cell (hESC) derived cardiovascular progenitor cells…”
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CRISPR-Mediated Genomic Addition to CPS1 Deficient iPSCs is Insufficient to Restore Nitrogen Homeostasis by Nitzahn, Matthew, Truong, Brian, Khoja, Suhail, Vega-Crespo, Agustin, Le, Colleen, Eliav, Adam, Makris, Georgios, Pyle, April D, Häberle, Johannes, Lipshutz, Gerald S

“…CPS1 deficiency is an inborn error of metabolism caused by loss-of-function mutations in the gene, catalyzing the initial reaction of the urea cycle…”
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Hepatic glutamine synthetase augmentation enhances ammonia detoxification by Soria, Leandro R., Nitzahn, Matthew, De Angelis, Angela, Khoja, Suhail, Attanasio, Sergio, Annunziata, Patrizia, Palmer, Donna J., Ng, Philip, Lipshutz, Gerald S., Brunetti‐Pierri, Nicola

“…The urea cycle and glutamine synthetase (GS) are the two main pathways for waste nitrogen removal and their deficiency results in hyperammonemia. Here, we…”
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An Improved Technique for Chromosomal Analysis of Human ES and iPS Cells by Moralli, Daniela, Yusuf, Mohammed, Mandegar, Mohammad A., Khoja, Suhail, Monaco, Zoia L., Volpi, Emanuela V.

“…Prolonged in vitro culture of human embryonic stem (hES) cells can result in chromosomal abnormalities believed to confer a selective advantage. This potential…”
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Hsv-1 amplicon system for human artificial chromosome formation in human es/ips cells and pluripotency induction by Khoja, Suhail

“…Development of safe and efficient approaches for gene delivery in human embryonic stem cells (hESc) and particularly in human induced pluripotent stem (hiPS)…”
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Evidence for a "Founder Effect" among HIV-infected injection drug users by Rai, Mohammad A, Nerurkar, Vivek R, Khoja, Suhail, Khan, Saeed, Yanagihara, Richard, Rehman, Arish, Kazmi, Shahana U, Ali, Syed H

“…We have previously reported a HIV-1 subtype A infection in a community of injection drug users (IDUs) in Karachi, Pakistan. We now show that this infection…”
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