A Transcriptome‐Based Drug Discovery Paradigm for Neurodevelopmental Disorders-Reference-Cited by-同舟云学术

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A Transcriptome‐Based Drug Discovery Paradigm for Neurodevelopmental Disorders

Published:2020-11-18 Issue:2 Volume:89 Page:199-211
ISSN:0364-5134
Container-title:Annals of Neurology
language:en
Short-container-title:Annals of Neurology

Author:

Dhindsa Ryan S.¹²^ORCID,Zoghbi Anthony W.¹³,Krizay Daniel K.¹⁴,Vasavda Chirag⁵^ORCID,Goldstein David B.¹⁴

Affiliation:

1. Institute for Genomic Medicine Columbia University Irving Medical Center New York NY USA

2. Integrated Program in Cellular, Molecular, and Biomedical Studies Columbia University Irving Medical Center New York NY USA

3. Department of Psychiatry Columbia University Irving Medical Center and New York State Psychiatric Institute New York NY USA

4. Department of Genetics & Development Columbia University Irving Medical Center New York NY USA

5. Solomon H. Snyder Department of Neuroscience Johns Hopkins University School of Medicine Baltimore MD USA

Publisher

Wiley

Subject

Neurology (clinical),Neurology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/ana.25950

Reference83 articles.

1. A brief history of human disease genetics

2. Genetic Discoveries Drive Molecular Analyses and Targeted Therapeutic Options in the Epilepsies

3. Enzyme replacement and enhancement therapies: lessons from lysosomal disorders

4. Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy

5. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

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1. Transcriptomic analysis identifies dysregulated pathways and therapeutic targets in PMM2-CDG;Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease;2024-06

2. Transcriptome-based screening in TARDBP/TDP-43 knock-in motor neurons identifies the NEDD8-activating enzyme inhibitor MLN4924;2024-01-31

3. Curbing Rhes Actions: Mechanism-based Molecular Target for Huntington’s Disease and Tauopathies;CNS & Neurological Disorders - Drug Targets;2024-01

4. Neurodevelopmental deficits and cell-type-specific transcriptomic perturbations in a mouse model of HNRNPU haploinsufficiency;PLOS Genetics;2023-10-02

5. Evidence of shared transcriptomic dysregulation of HNRNPU-related disorder between human organoids and embryonic mice;iScience;2023-01

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