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Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell lines.
Baxi, Emily G; Thompson, Terri; Li, Jonathan; Kaye, Julia A; Lim, Ryan G; Wu, Jie; Ramamoorthy, Divya; Lima, Leandro; Vaibhav, Vineet; Matlock, Andrea; Frank, Aaron; Coyne, Alyssa N; Landin, Barry; Ornelas, Loren; Mosmiller, Elizabeth; Thrower, Sara; Farr, S Michelle; Panther, Lindsey; Gomez, Emilda; Galvez, Erick; Perez, Daniel; Meepe, Imara; Lei, Susan; Mandefro, Berhan; Trost, Hannah; Pinedo, Louis; Banuelos, Maria G; Liu, Chunyan; Moran, Ruby; Garcia, Veronica; Workman, Michael; Ho, Richie; Wyman, Stacia; Roggenbuck, Jennifer; Harms, Matthew B; Stocksdale, Jennifer; Miramontes, Ricardo; Wang, Keona; Venkatraman, Vidya; Holewenski, Ronald; Sundararaman, Niveda; Pandey, Rakhi; Manalo, Danica-Mae; Donde, Aneesh; Huynh, Nhan; Adam, Miriam; Wassie, Brook T; Vertudes, Edward; Amirani, Naufa; Raja, Krishna.
Afiliação
  • Baxi EG; Brain Science Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
  • Thompson T; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
  • Li J; On Point Scientific Inc., San Diego, CA, USA.
  • Kaye JA; Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Lim RG; Center for Systems and Therapeutics and the Taube/Koret Center for Neurodegenerative Disease, Gladstone Institutes and the Departments of Neurology and Physiology, University of California, San Francisco, San Francisco, CA, USA.
  • Wu J; UCI MIND, University of California, Irvine, CA, USA.
  • Ramamoorthy D; Department of Biological Chemistry, University of California, Irvine, CA, USA.
  • Lima L; Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Vaibhav V; Center for Systems and Therapeutics and the Taube/Koret Center for Neurodegenerative Disease, Gladstone Institutes and the Departments of Neurology and Physiology, University of California, San Francisco, San Francisco, CA, USA.
  • Matlock A; Advanced Clinical Biosystems Research Institute, The Barbra Streisand Heart Center, The Smidt Heart Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Frank A; Advanced Clinical Biosystems Research Institute, The Barbra Streisand Heart Center, The Smidt Heart Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Coyne AN; Cedars-Sinai Biomanufacturing Center, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Landin B; Brain Science Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
  • Ornelas L; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
  • Mosmiller E; Computational Biology Center, IBM T.J. Watson Research Center, Yorktown Heights, NY, USA.
  • Thrower S; Cedars-Sinai Biomanufacturing Center, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Farr SM; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
  • Panther L; Department of Neurology, Healey Center, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
  • Gomez E; Technome LLC, Herndon, VA, USA.
  • Galvez E; Cedars-Sinai Biomanufacturing Center, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Perez D; Cedars-Sinai Biomanufacturing Center, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Meepe I; Cedars-Sinai Biomanufacturing Center, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Lei S; Cedars-Sinai Biomanufacturing Center, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Mandefro B; Cedars-Sinai Biomanufacturing Center, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Trost H; Cedars-Sinai Biomanufacturing Center, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Pinedo L; The Board of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Banuelos MG; The Board of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Liu C; Cedars-Sinai Biomanufacturing Center, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Moran R; The Board of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Garcia V; Cedars-Sinai Biomanufacturing Center, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Workman M; Cedars-Sinai Biomanufacturing Center, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Ho R; The Board of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Wyman S; The Board of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Roggenbuck J; The Board of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Harms MB; Center for Systems and Therapeutics and the Taube/Koret Center for Neurodegenerative Disease, Gladstone Institutes and the Departments of Neurology and Physiology, University of California, San Francisco, San Francisco, CA, USA.
  • Stocksdale J; Zofia Consulting, Reston, VA, USA.
  • Miramontes R; Department of Neurology and Genetics, Ohio State University Wexner Medical Center, Columbus, OH, USA.
  • Wang K; Department of Psychiatry and Human Behavior and Sue and Bill Gross Stem Cell Center, University of California, Irvine, CA, USA.
  • Venkatraman V; UCI MIND, University of California, Irvine, CA, USA.
  • Holewenski R; Department of Psychiatry and Human Behavior and Sue and Bill Gross Stem Cell Center, University of California, Irvine, CA, USA.
  • Sundararaman N; Advanced Clinical Biosystems Research Institute, The Barbra Streisand Heart Center, The Smidt Heart Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Pandey R; Advanced Clinical Biosystems Research Institute, The Barbra Streisand Heart Center, The Smidt Heart Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Manalo DM; Advanced Clinical Biosystems Research Institute, The Barbra Streisand Heart Center, The Smidt Heart Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Donde A; Advanced Clinical Biosystems Research Institute, The Barbra Streisand Heart Center, The Smidt Heart Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Huynh N; Advanced Clinical Biosystems Research Institute, The Barbra Streisand Heart Center, The Smidt Heart Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
  • Adam M; Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Wassie BT; Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Vertudes E; Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Amirani N; Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Raja K; Center for Systems and Therapeutics and the Taube/Koret Center for Neurodegenerative Disease, Gladstone Institutes and the Departments of Neurology and Physiology, University of California, San Francisco, San Francisco, CA, USA.
Nat Neurosci ; 25(2): 226-237, 2022 02.
Article em En | MEDLINE | ID: mdl-35115730
ABSTRACT
Answer ALS is a biological and clinical resource of patient-derived, induced pluripotent stem (iPS) cell lines, multi-omic data derived from iPS neurons and longitudinal clinical and smartphone data from over 1,000 patients with ALS. This resource provides population-level biological and clinical data that may be employed to identify clinical-molecular-biochemical subtypes of amyotrophic lateral sclerosis (ALS). A unique smartphone-based system was employed to collect deep clinical data, including fine motor activity, speech, breathing and linguistics/cognition. The iPS spinal neurons were blood derived from each patient and these cells underwent multi-omic analytics including whole-genome sequencing, RNA transcriptomics, ATAC-sequencing and proteomics. The intent of these data is for the generation of integrated clinical and biological signatures using bioinformatics, statistics and computational biology to establish patterns that may lead to a better understanding of the underlying mechanisms of disease, including subgroup identification. A web portal for open-source sharing of all data was developed for widespread community-based data analytics.
Assuntos

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Células-Tronco Pluripotentes Induzidas / Esclerose Lateral Amiotrófica Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Células-Tronco Pluripotentes Induzidas / Esclerose Lateral Amiotrófica Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article