Faculty profile
Wei-Hsiang Huang
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Research
Latest papers
CRISPR screening identifies TRIM27 as a destabilizer of the Smith-Magenis syndrome protein RAI1.
Genetics · 2026 · senior author
Molecular and developmental deficits in Smith-Magenis syndrome human stem cell-derived cortical neural models.
American journal of human genetics · 2025 · senior author
Tunable, proteolytic dosage control of CRISPR-Cas systems enables precise gene therapy for dosage sensitive disorders.
bioRxiv : the preprint server for biology · 2025
Latest funding
- $30,000
Molecular pathogenesis of Smith-Magenis syndrome and developing therapy using hiPSC-derived neuronal models
CIHR · 2025 · Nominated PI
- $1,025,100
Molecular pathogenesis of Smith-Magenis syndrome and developing therapy using hiPSC-derived neuronal models
CIHR · 2025 · Nominated PI
- $150,000
Mechanistic investigation of how cortical clonal mutation drives brain-wide developmental defects
CIHR · 2025 · Nominated PI
12 publications.
CRISPR screening identifies TRIM27 as a destabilizer of the Smith-Magenis syndrome protein RAI1.
Lin YC, Lee YJ, Li CX, Chang HC, Kowalczyk M, Haque M, Dragoiescu A, Losier TT, Taylor JA, Rousseaux MWC, Huang WH
Molecular and developmental deficits in Smith-Magenis syndrome human stem cell-derived cortical neural models.
Lee YJ, Chang YT, Cho Y, Kowalczyk M, Dragoiescu A, Pacis A, Kailasam S, Lefebvre F, Zhang Q, Gao X, Huang WH
Tunable, proteolytic dosage control of CRISPR-Cas systems enables precise gene therapy for dosage sensitive disorders.
Katz N, An C, Lee YJ, Tycko J, Zhang M, Kang J, Bintu L, Bassik MC, Huang WH, Gao XJ
TSC2-mTORC1 axis regulates morphogenesis and neurological function of Gli1+ adult-born dentate granule cells.
Kowalczyk M, Lee YJ, Huang WH
Comparative analyses of the Smith-Magenis syndrome protein RAI1 in mice and common marmoset monkeys.
Chang YT, Lee YJ, Haque M, Chang HC, Javed S, Lin YC, Cho Y, Abramovitz J, Chin G, Khamis A, Raja R, Murai KK, Huang WH
Smith-Magenis syndrome protein RAI1 regulates body weight homeostasis through hypothalamic BDNF-producing neurons and neurotrophin downstream signalling.
Javed S, Chang YT, Cho Y, Lee YJ, Chang HC, Haque M, Lin YC, Huang WH
rAAV-CRISPRa therapy corrects Rai1 haploinsufficiency and rescues selective disease features in Smith-Magenis syndrome mice.
Chang HC, Lee YJ, Javed S, Haque M, Chang YT, Lin YC, Oram C, Huang WH
Loss of Rai1 enhances hippocampal excitability and epileptogenesis in mouse models of Smith-Magenis syndrome.
Chang YT, Kowalczyk M, Fogerson PM, Lee YJ, Haque M, Adams EL, Wang DC, DeNardo LA, Tessier-Lavigne M, Huguenard JR, Luo L, Huang WH
Performing Single-Cell Clonal Analysis in the Mouse Brain Using Mosaic Analysis with Double Markers (MADM).
Huang WH
Temporal dissection of Rai1 function reveals brain-derived neurotrophic factor as a potential therapeutic target for Smith-Magenis syndrome.
Javed S, Lee YJ, Xu J, Huang WH
Molecular pathogenesis of Smith-Magenis syndrome and developing therapy using hiPSC-derived neuronal models
Principal investigators: Huang, Wei-Hsiang
Keywords: Hipsc-Derived Neuronal Models; Molecular Pathogenesis; Smith-Magenis Syndrome
Molecular pathogenesis of Smith-Magenis syndrome and developing therapy using hiPSC-derived neuronal models
Principal investigators: Huang, Wei-Hsiang
Keywords: Autism; Cortical Organoid; Electrophysiology; Hipsc-Derived Neuron; Induced Pluripotent Stem Cell (Hipsc); Neural Progenitor Cells; Single Cell Rna-Sequencing
Mechanistic investigation of how cortical clonal mutation drives brain-wide developmental defects
Principal investigators: Huang, Wei-Hsiang
Keywords: Brain Development; Mosaic Analysis; Neurodevelopmental Disorder; Single-Cell Transcriptomics
Molecular pathogenesis of Smith-Magenis syndrome and developing therapy using hiPSC-derived neuronal models
Principal investigators: Huang, Wei-Hsiang
Keywords: Autism; Cortical Organoid; Electrophysiology; Hipsc-Derived Neuron; Induced Pluripotent Stem Cell (Hipsc); Neurodevelopment
Creating an integrated platform for in vivo brain targeting and experimentation
Principal investigators: Sabatini, Paul
Mechanism and Treatment for Neuronal Hyperexcitability in Smith-Magenis Syndrome
Principal investigators: Huang, Wei-Hsiang
Keywords: Genetic Disorders; Ion Channel; Mouse Behavior; Neurodevelopmental Disorders; Neuronal Excitability
The role of Retinoic Acid Induced 1 (Rai1) in neural network stability
Principal investigators: Huang, Wei-Hsiang
Determine how somatic gene deletion causes disordered childhood brain development
Principal investigators: Huang, Wei-Hsiang
Keywords: .
The role of Retinoic Acid Induced 1 (Rai1) in neural network stability
Principal investigators: Huang, Wei-Hsiang
From CIHR, NSERC and SSHRC funding decisions: CIHR since 2008, NSERC since 1991 and SSHRC since 1998, including their latest published competition results.
Frequent collaborators
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