Works matching AU Jafar-nejad, Paymaan

Results: 33

Scn8a Antisense Oligonucleotide Is Protective in Mouse Models of SCN8A Encephalopathy and Dravet Syndrome.

Published in:

2020

By:

Lenk, Guy M.;
Jafar‐Nejad, Paymaan;
Hill, Sophie F.;
Huffman, Lucas D.;
Smolen, Corrine E.;
Wagnon, Jacy L.;
Petit, Hayley;
Yu, Wenxi;
Ziobro, Julie;
Bhatia, Kritika;
Parent, Jack;
Giger, Roman J.;
Rigo, Frank;
Meisler, Miriam H.;
Jafar-Nejad, Paymaan

Publication type:

journal article

Targeting mechanistic target of rapamycin complex 2 attenuates immunopathology in systemic lupus erythematosus Open Access.

Published in:

Rheumatology, 2025, v. 64, n. 6, p. 3964, doi. 10.1093/rheumatology/keae662

By:

Ai, Minji;
Zhou, Xian;
Carrer, Michele;
Jafar-nejad, Paymaan;
Li, Yanfeng;
Gades, Naomi;
Alexander, Mariam;
Bautista-Vargas, Mario;
Duarte-García, Alí;
Zeng, Hu

Publication type:

Article

Duplication of Atxn1l suppresses SCA1 neuropathology by decreasing incorporation of polyglutamine-expanded ataxin-1 into native complexes.

Published in:

Nature Genetics, 2007, v. 39, n. 3, p. 373, doi. 10.1038/ng1977

By:

Bowman, Aaron B.;
Lam, Yung C.;
Jafar-Nejad, Paymaan;
Hung-Kai Chen;
Richman, Ronald;
Samaco, Rodney C.;
Fryer, John D.;
Kahle, Juliette J.;
Orr, Harry T.;
Zoghbi, Huda Y.

Publication type:

Article

Loss of CHOP Prevents Joint Degeneration and Pain in a Mouse Model of Pseudoachondroplasia.

Published in:

International Journal of Molecular Sciences, 2025, v. 26, n. 1, p. 16, doi. 10.3390/ijms26010016

By:

Hecht, Jacqueline T.;
Veerisetty, Alka C.;
Hossain, Mohammad G.;
Patra, Debabrata;
Carrer, Michele;
Chiu, Frankie;
Relic, Dorde;
Jafar-nejad, Paymaan;
Posey, Karen L.

Publication type:

Article

Pharmacometabolomic Signature of Ataxia SCA1 Mouse Model and Lithium Effects.

Published in:

PLoS ONE, 2013, v. 8, n. 8, p. 1, doi. 10.1371/journal.pone.0070610

By:

Perroud, Bertrand;
Jafar-Nejad, Paymaan;
Wikoff, William R.;
Gatchel, Jennifer R.;
Wang, Lu;
Barupal, Dinesh K.;
Crespo-Barreto, Juan;
Fiehn, Oliver;
Zoghbi, Huda Y.;
Kaddurah-Daouk, Rima

Publication type:

Article

Longitudinal EEG model detects antisense oligonucleotide treatment effect and increased UBE3A in Angelman syndrome.

Published in:

Brain Communications, 2022, v. 4, n. 3, p. 1, doi. 10.1093/braincomms/fcac106

By:

Spencer, Elizabeth R.;
Wen Shi;
Komorowski, Robert W.;
Gilbert, James P.;
Ostrowski, Lauren M.;
Bird, Lynne M.;
Thibert, Ronald;
Bao, Channa;
Molloy, Fiona;
Calhoun, Michael;
Koirala, Samir;
Jafar-nejad, Paymaan;
Rigo, Frank;
Kramer, Mark A.;
Chu, Catherine J.

Publication type:

Article

Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease.

Published in:

Clinical & Translational Medicine, 2025, v. 15, n. 4, p. 1, doi. 10.1002/ctm2.70314

By:

Weiss, Lan;
Carrer, Michele;
Shmara, Alyaa;
Martin, Angela;
Yin, Hong;
Pal, Pallabi;
Cheng, Cheng;
Ta, Lac;
Boock, Victoria;
Fazeli, Yasamin;
Chang, Mindy;
Paguio, Marvin;
Lee, Jonathan;
Yu, Howard;
Weiss, John;
Grossman, Tamar R;
Raben, Nina;
Jafar‐Nejad, Paymaan;
Kimonis, Virginia

Publication type:

Article

Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome.

Published in:

Science Translational Medicine, 2021, v. 13, n. 583, p. 1, doi. 10.1126/scitranslmed.aaz7785

By:

Shao, Yingyao;
Sztainberg, Yehezkel;
Wang, Qi;
Bajikar, Sameer S.;
Trostle, Alexander J.;
Wan, Ying-Wooi;
Jafar-nejad, Paymaan;
Rigo, Frank;
Liu, Zhandong;
Tang, Jianrong;
Zoghbi, Huda Y.

Publication type:

Article

Frontotemporal dementia causative CHMP2B impairs neuronal endolysosomal traffic-rescue by TMEM106B knockdown.

Published in:

2018

By:

Clayton, Emma L;
Milioto, Carmelo;
Muralidharan, Bhavana;
Norona, Frances E;
Edgar, James R;
Soriano, Armand;
Jafar-nejad, Paymaan;
Rigo, Frank;
Collinge, John;
Isaacs, Adrian M

Publication type:

journal article

Antisense oligonucleotides targeting miR‐29b binding site increase translation of progranulin protein: potential therapeutic strategy for progranulin‐deficient frontotemporal dementia.

Published in:

Alzheimer's & Dementia: The Journal of the Alzheimer's Association, 2022, v. 18, n. 10, p. 1, doi. 10.1002/alz.067828

By:

Aggarwal, Geetika;
Banerjee, Subhashis;
Jones, Spencer A.;
Pavlack, Monica;
Benchaar, Yousri;
Bélanger, Jasmine;
Sévigny, Myriam;
Smith, Denise M.;
Niehoff, Michael L.;
de Vera, Ian Mitchelle S.;
Petkau, Terri L.;
Leavitt, Blair R.;
Ling, Karen;
Jafar‐nejad, Paymaan;
Rigo, Frank;
Morley, John E.;
Farr, Susan A.;
Dutchak, Paul A.;
Sephton, Chantelle F.;
Nguyen, Andrew D.

Publication type:

Article

O4‐02‐02: TARGETING THE ALZHEIMER'S DISEASE‐ASSOCIATED RISK GENE, TREM2, WITH ANTISENSE OLIGONUCLEOTIDES.

Published in:

Alzheimer's & Dementia: The Journal of the Alzheimer's Association, 2018, v. 14, p. P1402, doi. 10.1016/j.jalz.2018.06.2916

By:

Schoch, Kathleen M.;
Jafar-nejad, Paymaan;
Rigo, Frank;
Miller, Timothy M.

Publication type:

Article

PFTK1 kinase regulates axogenesis during development via RhoA activation.

Published in:

BMC Biology, 2023, v. 21, n. 1, p. 1, doi. 10.1186/s12915-023-01732-w

By:

González, Yasmilde Rodríguez;
Kamkar, Fatemeh;
Jafar-nejad, Paymaan;
Wang, Suzi;
Qu, Dianbo;
Alvarez, Leticia Sanchez;
Hawari, Dina;
Sonnenfeld, Margaret;
Slack, Ruth S.;
Albert, Paul R.;
Park, David S.;
Joselin, Alvin

Publication type:

Article

Molecular Profiling of Mouse Models of Loss or Gain of Function of the KCNT1 (Slack) Potassium Channel and Antisense Oligonucleotide Treatment.

Published in:

Biomolecules (2218-273X), 2024, v. 14, n. 11, p. 1397, doi. 10.3390/biom14111397

By:

Sun, Fangxu;
Wang, Huafeng;
Wu, Jing;
Quraishi, Imran H.;
Zhang, Yalan;
Pedram, Maysam;
Gao, Benbo;
Jonas, Elizabeth A.;
Nguyen, Viet;
Wu, Sijia;
Mabrouk, Omar S.;
Jafar-nejad, Paymaan;
Kaczmarek, Leonard K.

Publication type:

Article

A NOTCH3 pathogenic variant influences osteogenesis and can be targeted by antisense oligonucleotides in induced pluripotent stem cells.

Published in:

PLoS ONE, 2025, v. 20, n. 1, p. 1, doi. 10.1371/journal.pone.0316644

By:

Canalis, Ernesto;
Yu, Jungeun;
Schilling, Lauren;
Jafar-nejad, Paymaan;
Carrer, Michele

Publication type:

Article

Reduction of Kcnt1 is therapeutic in mouse models of SCN1A and SCN8A epilepsy.

Published in:

Frontiers in Neuroscience, 2023, p. 01, doi. 10.3389/fnins.2023.1282201

By:

Hill, Sophie F.;
Jafar-Nejad, Paymaan;
Rigo, Frank;
Meisler, Miriam H.

Publication type:

Article

Modeling antisense oligonucleotide therapy in MECP2 duplication syndrome human iPSC-derived neurons reveals gene expression programs responsive to MeCP2 levels.

Published in:

Human Molecular Genetics, 2024, v. 33, n. 22, p. 1986, doi. 10.1093/hmg/ddae135

By:

Bajikar, Sameer S;
Sztainberg, Yehezkel;
Trostle, Alexander J;
Tirumala, Harini P;
Wan, Ying-Wooi;
Harrop, Caroline L;
Bengtsson, Jesse D;
Carvalho, Claudia M B;
Pehlivan, Davut;
Suter, Bernhard;
Neul, Jeffrey L;
Liu, Zhandong;
Jafar-Nejad, Paymaan;
Rigo, Frank;
Zoghbi, Huda Y

Publication type:

Article

Targeting nonsense-mediated RNA decay does not increase progranulin levels in the GrnR493X mouse model of frontotemporal dementia.

Published in:

PLoS ONE, 2023, v. 18, n. 3, p. 1, doi. 10.1371/journal.pone.0282822

By:

Smith, Denise M.;
Niehoff, Michael L.;
Ling, Karen;
Jafar-Nejad, Paymaan;
Rigo, Frank;
Farr, Susan A.;
Wilkinson, Miles F.;
Nguyen, Andrew D.

Publication type:

Article

Phosphorylation of ATXN1 at Ser776 in the cerebellum.

Published in:

Journal of Neurochemistry, 2009, v. 110, n. 2, p. 675, doi. 10.1111/j.1471-4159.2009.06164.x

By:

Jorgensen, Nathan D.;
Andresen, J. Michael;
Lagalwar, Sara;
Armstrong, Ben;
Stevens, Sam;
Byam, Courtney E.;
Duvick, Lisa A.;
Shaojuan Lai;
Jafar-Nejad, Paymaan;
Zoghbi, Huda Y.;
Clark, H. Brent;
Orr, Harry T.

Publication type:

Article

Antisense oligonucleotide therapy rescues disturbed brain rhythms and sleep in juvenile and adult mouse models of Angelman syndrome.

Published in:

eLife, 2023, p. 1, doi. 10.7554/eLife.81892

By:

Dongwon Lee;
Wu Chen;
Kaku, Heet Naresh;
Xinming Zhuo;
Chao, Eugene S.;
Soriano, Armand;
Kuncheria, Allen;
Flores, Stephanie;
Joo Hyun Kim;
Rivera, Armando;
Rigo, Frank;
Jafar-nejad, Paymaan;
Beaudet, Arthur L.;
Caudill, Matthew S.;
Mingshan Xue

Publication type:

Article

The differential impact of HNRNPA1 isoforms on gene expression and their relevance to dsRNA-mediated innate immune response.

Published in:

Scientific Reports, 2025, v. 15, n. 1, p. 1, doi. 10.1038/s41598-025-99031-7

By:

Deshaies, Jade-Emmanuelle;
Triassi, Valérie;
Lacombe, Andréanne;
Gagné, Myriam;
Ling, Karen;
Ghosh, Asmita;
Labrecque, Marjorie;
Rigo, Frank;
Jafar-nejad, Paymaan;
Tétreault, Martine;
Vande Velde, Christine

Publication type:

Article

Conjugation to a transferrin receptor 1-binding Bicycle peptide enhances ASO and siRNA potency in skeletal and cardiac muscles Open Access.

Published in:

Nucleic Acids Research, 2025, v. 53, n. 7, p. 1, doi. 10.1093/nar/gkaf270

By:

Østergaard, Michael E;
Carrer, Michele;
Anderson, Brooke A;
Afetian, Megan;
Bakooshli, Mohsen A;
Santos, Jinro A;
Klein, Stephanie K;
Capitanio, Juliana;
Freestone, Graeme C;
Tanowitz, Michael;
Galindo-Murillo, Rodrigo;
Gaus, Hans J;
Dwyer, Chrissa A;
Jackson, Michaela;
Jafar-nejad, Paymaan;
Rigo, Frank;
Seth, Punit P;
Gaynor, Katherine U;
Stanway, Steven J;
Urbonas, Liudvikas

Publication type:

Article

The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration.

Published in:

Nucleic Acids Research, 2021, v. 49, n. 2, p. 657, doi. 10.1093/nar/gkaa1235

By:

Jafar-nejad, Paymaan;
Powers, Berit;
Soriano, Armand;
Zhao, Hien;
Norris, Daniel A;
Matson, John;
DeBrosse-Serra, Beatrice;
Watson, Jamie;
Narayanan, Padmakumar;
Chun, Seung J;
Mazur, Curt;
Kordasiewicz, Holly;
Swayze, Eric E;
Rigo, Frank

Publication type:

Article

Opposing effects of polyglutamine expansion on native protein complexes contribute to SCA1.

Published in:

Nature, 2008, v. 452, n. 7188, p. 713, doi. 10.1038/nature06731

By:

Janghoo Lim;
Crespo-Barreto, Juan;
Jafar-Nejad, Paymaan;
Bowman, Aaron B.;
Richman, Ronald;
Hill, David E.;
Orr, Harry T.;
Zoghbi, Huda Y.

Publication type:

Article

Genetic Downregulation of the Metabotropic Glutamate Receptor Type 5 Dampens the Reactive and Neurotoxic Phenotype of Adult ALS Astrocytes.

Published in:

Cells (2073-4409), 2023, v. 12, n. 15, p. 1952, doi. 10.3390/cells12151952

By:

Torazza, Carola;
Provenzano, Francesca;
Gallia, Elena;
Cerminara, Maria;
Balbi, Matilde;
Bonifacino, Tiziana;
Tessitore, Sara;
Ravera, Silvia;
Usai, Cesare;
Musante, Ilaria;
Puliti, Aldamaria;
Van Den Bosch, Ludo;
Jafar-nejad, Paymaan;
Rigo, Frank;
Milanese, Marco;
Bonanno, Giambattista

Publication type:

Article

Carbogen-Induced Respiratory Acidosis Blocks Experimental Seizures by a Direct and Specific Inhibition of NaV1.2 Channels in the Axon Initial Segment of Pyramidal Neurons.

Published in:

Journal of Neuroscience, 2023, v. 43, n. 10, p. 1658, doi. 10.1523/JNEUROSCI.1387-22.2022

By:

Hatch, Robert J.;
Berecki, Géza;
Jancovski, Nikola;
Li, Melody;
Rollo, Ben;
Jafar-Nejad, Paymaan;
Rigo, Frank;
Kaila, Kai;
Reid, Christopher A.;
Petrou, Steven

Publication type:

Article

Targeted suppression of mTORC2 reduces seizures across models of epilepsy.

Published in:

Nature Communications, 2023, v. 14, n. 1, p. 1, doi. 10.1038/s41467-023-42922-y

By:

Okoh, James;
Mays, Jacqunae;
Bacq, Alexandre;
Oses-Prieto, Juan A.;
Tyanova, Stefka;
Chen, Chien-Ju;
Imanbeyev, Khalel;
Doladilhe, Marion;
Zhou, Hongyi;
Jafar-Nejad, Paymaan;
Burlingame, Alma;
Noebels, Jeffrey;
Baulac, Stephanie;
Costa-Mattioli, Mauro

Publication type:

Article

NMNAT2 supports vesicular glycolysis via NAD homeostasis to fuel fast axonal transport.

Published in:

Molecular Neurodegeneration, 2024, v. 19, n. 1, p. 1, doi. 10.1186/s13024-023-00690-9

By:

Yang, Sen;
Niou, Zhen-Xian;
Enriquez, Andrea;
LaMar, Jacob;
Huang, Jui-Yen;
Ling, Karen;
Jafar-Nejad, Paymaan;
Gilley, Jonathan;
Coleman, Michael P.;
Tennessen, Jason M.;
Rangaraju, Vidhya;
Lu, Hui-Chen

Publication type:

Article

Genetic interaction between Scn8a and potassium channel genes Kcna1 and Kcnq2.

Published in:

Epilepsia (Series 4), 2022, v. 63, n. 10, p. e125, doi. 10.1111/epi.17374

By:

Hill, Sophie F.;
Ziobro, Julie M.;
Jafar‐Nejad, Paymaan;
Rigo, Frank;
Meisler, Miriam H.

Publication type:

Article

G2C4 targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS/FTD induced pluripotent stem cell derived neurons.

Published in:

Acta Neuropathologica, 2024, v. 147, n. 1, p. 1, doi. 10.1007/s00401-023-02652-3

By:

Rothstein, Jeffrey D.;
Baskerville, Victoria;
Rapuri, Sampath;
Mehlhop, Emma;
Jafar-Nejad, Paymaan;
Rigo, Frank;
Bennett, Frank;
Mizielinska, Sarah;
Isaacs, Adrian;
Coyne, Alyssa N.

Publication type:

Article

RAS-MAPK-MSK1 pathway modulates ataxin 1 protein levels and toxicity in SCA1.

Published in:

Nature, 2013, v. 498, n. 7454, p. 325, doi. 10.1038/nature12204

By:

Park, Jeehye;
Al-Ramahi, Ismael;
Tan, Qiumin;
Mollema, Nissa;
Diaz-Garcia, Javier R.;
Gallego-Flores, Tatiana;
Lu, Hsiang-Chih;
Lagalwar, Sarita;
Duvick, Lisa;
Kang, Hyojin;
Lee, Yoontae;
Jafar-Nejad, Paymaan;
Sayegh, Layal S.;
Richman, Ronald;
Liu, Xiuyun;
Gao, Yan;
Shaw, Chad A.;
Arthur, J. Simon C.;
Orr, Harry T.;
Westbrook, Thomas F.

Publication type:

Article

TRIM28 regulates the nuclear accumulation and toxicity of both alpha- synuclein and tau.

Published in:

eLife, 2016, p. 1, doi. 10.7554/eLife.19809

By:

Rousseaux, Maxime W. C.;
de Haro, Maria;
Lasagna-Reeves, Cristian A.;
De Maio, Antonia;
Park, Jeehye;
Jafar-Nejad, Paymaan;
Al-Ramahi, Ismael;
Sharma, Ajay;
See, Lauren;
Lu, Nan;
Vilanova-Velez, Luis;
Klisch, Tiemo J.;
Westbrook, Thomas F.;
Troncoso, Juan C.;
Botas, Juan;
Zoghbi, Huda Y.

Publication type:

Article

Ataxin-1 oligomers induce local spread of pathology and decreasing them by passive immunization slows Spinocerebellar ataxia type 1 phenotypes.

Published in:

eLife, 2015, p. 1, doi. 10.7554/eLife.10891

By:

Lasagna-Reeves, Cristian A.;
Rousseaux, Maxime W. C.;
Guerrero-Munoz, Marcos J.;
Vilanova-Velez, Luis;
Park, Jeehye;
See, Lauren;
Jafar-Nejad, Paymaan;
Richman, Ronald;
Orr, Harry T.;
Kayed, Rakez;
Zoghbi, Huda Y.

Publication type:

Article

A native interactor scaffolds and stabilizes toxic ATAXIN-1 oligomers in SCA1.

Published in:

eLife, 2015, p. 1, doi. 10.7554/eLife.07558

By:

Lasagna-Reeves, Cristian A.;
Rousseaux, Maxime W. C.;
Guerrero Muñoz, Marcos J.;
Jeehye Park;
Jafar-Nejad, Paymaan;
Richman, Ronald;
Nan Lu;
Sengupta, Urmi;
Litvinchuk, Alexandra;
Orr, Harry T.;
Kayed, Rakez;
Zoghbi, Huda Y.

Publication type:

Article

Works matching AU Jafar-nejad, Paymaan

Scn8a Antisense Oligonucleotide Is Protective in Mouse Models of SCN8A Encephalopathy and Dravet Syndrome.

Targeting mechanistic target of rapamycin complex 2 attenuates immunopathology in systemic lupus erythematosus Open Access.

Duplication of Atxn1l suppresses SCA1 neuropathology by decreasing incorporation of polyglutamine-expanded ataxin-1 into native complexes.

Loss of CHOP Prevents Joint Degeneration and Pain in a Mouse Model of Pseudoachondroplasia.

Pharmacometabolomic Signature of Ataxia SCA1 Mouse Model and Lithium Effects.

Longitudinal EEG model detects antisense oligonucleotide treatment effect and increased UBE3A in Angelman syndrome.

Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease.

Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome.

Frontotemporal dementia causative CHMP2B impairs neuronal endolysosomal traffic-rescue by TMEM106B knockdown.

Antisense oligonucleotides targeting miR‐29b binding site increase translation of progranulin protein: potential therapeutic strategy for progranulin‐deficient frontotemporal dementia.

O4‐02‐02: TARGETING THE ALZHEIMER'S DISEASE‐ASSOCIATED RISK GENE, TREM2, WITH ANTISENSE OLIGONUCLEOTIDES.

PFTK1 kinase regulates axogenesis during development via RhoA activation.

Molecular Profiling of Mouse Models of Loss or Gain of Function of the KCNT1 (Slack) Potassium Channel and Antisense Oligonucleotide Treatment.

A NOTCH3 pathogenic variant influences osteogenesis and can be targeted by antisense oligonucleotides in induced pluripotent stem cells.

Reduction of Kcnt1 is therapeutic in mouse models of SCN1A and SCN8A epilepsy.

Modeling antisense oligonucleotide therapy in MECP2 duplication syndrome human iPSC-derived neurons reveals gene expression programs responsive to MeCP2 levels.

Targeting nonsense-mediated RNA decay does not increase progranulin levels in the Grn<sup>R493X</sup> mouse model of frontotemporal dementia.

Phosphorylation of ATXN1 at Ser776 in the cerebellum.

Antisense oligonucleotide therapy rescues disturbed brain rhythms and sleep in juvenile and adult mouse models of Angelman syndrome.

The differential impact of HNRNPA1 isoforms on gene expression and their relevance to dsRNA-mediated innate immune response.

Conjugation to a transferrin receptor 1-binding Bicycle peptide enhances ASO and siRNA potency in skeletal and cardiac muscles Open Access.

The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration.

Opposing effects of polyglutamine expansion on native protein complexes contribute to SCA1.

Genetic Downregulation of the Metabotropic Glutamate Receptor Type 5 Dampens the Reactive and Neurotoxic Phenotype of Adult ALS Astrocytes.

Carbogen-Induced Respiratory Acidosis Blocks Experimental Seizures by a Direct and Specific Inhibition of NaV1.2 Channels in the Axon Initial Segment of Pyramidal Neurons.

Targeted suppression of mTORC2 reduces seizures across models of epilepsy.

NMNAT2 supports vesicular glycolysis via NAD homeostasis to fuel fast axonal transport.

Genetic interaction between Scn8a and potassium channel genes Kcna1 and Kcnq2.

G<sub>2</sub>C<sub>4</sub> targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS/FTD induced pluripotent stem cell derived neurons.

RAS-MAPK-MSK1 pathway modulates ataxin 1 protein levels and toxicity in SCA1.

TRIM28 regulates the nuclear accumulation and toxicity of both alpha- synuclein and tau.

Ataxin-1 oligomers induce local spread of pathology and decreasing them by passive immunization slows Spinocerebellar ataxia type 1 phenotypes.

A native interactor scaffolds and stabilizes toxic ATAXIN-1 oligomers in SCA1.