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Timothy Yu

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Associate Member

Broad Institute

United States

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Professors in United States

Recent Grants

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Expert Panel: Genetics of Malformations of the Central Nervous System

Open Date: 2017-09-12

Close Date: 2020-08-31

Grant: Close

Complete gene knockouts in autism: identification and functional characterization

Open Date: 2017-07-21

Close Date: 2022-04-30

Articles (20)

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Collaborators (12)

Francesca Cogliati

IRCCS Istituto Auxologico Italiano

ITALY

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Abdulrahman Aldeeri

Harvard University

UNITED STATES

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Jonathan K. Watts

Professor

University of Massachusetts Chan Medical School

UNITED STATES

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Annemieke Aartsma-Rus

Professor of Translational Genetics

Leiden University Medical Center

NETHERLANDS

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Jo Gleeson

UC San Diego Health System

UNITED STATES

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Annapurna Poduri

Professor

Harvard University

UNITED STATES

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Richard Smith

Assistant Professor

Northwestern University Feinberg School of Medicine

UNITED STATES

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Thomas Kitzler

Scientist

Research Institute of the McGill University Health Centre

CANADA

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Florence Petit

Université de Lille

FRANCE

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Youjin Oh

Assistant Professor

University of Alberta

CANADA

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Ashley Hurst

Harvard University

UNITED STATES

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Junseok Park

Assistant Professor

Baylor College of Medicine

UNITED STATES

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Timothy Yu

Research Interests

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Recent Grants

Articles (20)

Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy

Atrophin-1 antisense oligonucleotide provides robust protection from pathology in a fully humanized DRPLA model

Response to the FDA's proposed pathway for individualized genetic therapies

Screening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: A retrospective cohort study

The Role of Academic Medical Centers in Personalized Experimental Therapeutic Development

The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing

Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments

Implementing customized genomic sequencing reports to empower providers in safety-net neonatal intensive care units

Multi-omic analysis of the ciliogenic transcription factor RFX3 reveals a role in promoting activity-dependent responses via enhancing CREB binding in human neurons

The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing (vol 53, gkaf346, 2025)

Deletions in the <i>CDKL5</i> 5′ untranslated region lead to CDKL5 deficiency disorder

<i>RPL26</i> variants: A rare cause of Diamond-Blackfan anemia syndrome with multiple congenital anomalies at the forefront

Epigenomic and phenotypic characterization of DEGCAGS syndrome

" It ' s hard to wait": " : Provider perspectives on current genomic care in safety-net NICUs

A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases

ATM-deficiency-induced microglial activation promotes neurodegeneration in ataxia-telangiectasia

How to pay for individualized genetic medicines

Implementation of rapid genomic sequencing in safety-net neonatal intensive care units: protocol for the VIrtual GenOme CenteR (VIGOR) proof-of-concept study

LETTER TO THE EDITOR Response to Horta et al

N-of-1 Studies in an Era of Precision Medicine

Collaborators (12)

Francesca Cogliati

Abdulrahman Aldeeri

Jonathan K. Watts

Annemieke Aartsma-Rus

Jo Gleeson

Annapurna Poduri

Richard Smith

Thomas Kitzler

Florence Petit

Youjin Oh

Ashley Hurst

Junseok Park

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