ArticleMolecular therapy. Methods & clinical development2014
Targeted CNS Delivery Using Human MiniPromoters and Demonstrated Compatibility with Adeno-Associated Viral Vectors.
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Article in Molecular therapy. Methods & clinical development, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 39 papers.
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39citing papers in PubMed
1.9field-weighted citation impact, top 15% of its field
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Who cites it
39 citing papers in PubMed, 57 citations in OpenAlex.
- An AAV-Based Therapy Approach for Neurological Phenotypes of X-Linked Adrenoleukodystrophy.International journal of molecular sciences · 2025Review
- MultiSite Assembly of Gateway Induced Clones (MAGIC): a flexible cloning toolbox for use in vertebrate model systems.Development (Cambridge, England) · 2025Article
- Gene therapy shines light on congenital stationary night blindness for future cures.Journal of translational medicine · 2025Review
- New MiniPromoter Ple389 (ADORA2A) drives selective expression in medium spiny neurons in mice and non-human primates.Scientific reports · 2024Article
- Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver.Gene therapy · 2024Article
- Engineering of the AAV-Compatible Hair Cell-Specific Small-Size Myo15 Promoter for Gene Therapy in the Inner Ear.Research (Washington, D.C.) · 2024Article
- Persistent transgene expression in peripheral tissues one year post intravenous and intramuscular administration of AAV vectors containing the alphaherpesvirus latency-associated promoter 2.Frontiers in virology (Lausanne, Switzerland) · 2024Article
- Article
- Spatial transcriptomics for profiling the tropism of viral vectors in tissues.Nature biotechnology · 2023Article
- OnTarget: in silico design of MiniPromoters for targeted delivery of expression.Nucleic acids research · 2023Article
- Developing New Vectors for Retinal Gene Therapy.Cold Spring Harbor perspectives in medicine · 2023Article
- Evaluating the transduction efficiency of systemically delivered AAV vectors in the rat nervous system.Frontiers in neuroscience · 2023Article
- Advanced genetic therapies for the treatment of Rett syndrome: state of the art and future perspectives.Frontiers in neuroscience · 2023Review
- Targeted Expression of Retinoschisin by Retinal Bipolar Cells in XLRS Promotes Resolution of Retinoschisis Cysts Sans RS1 From Photoreceptors.Investigative ophthalmology & visual science · 2022Article
- A systematic comparison of optogenetic approaches to visual restoration.Molecular therapy. Methods & clinical development · 2022Article
- Insulin-like growth factor I mitigates post-traumatic stress by inhibiting AMP-kinase in orexin neurons.Molecular psychiatry · 2022Article
- AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset.Nature neuroscience · 2022Article
- Intracerebroventricular Administration of AAV9-PHP.B SYN1-EmGFP Induces Widespread Transgene Expression in the Mouse and Monkey Central Nervous System.Human gene therapy · 2021Article
- ON-bipolar cell gene expression during retinal degeneration: Implications for optogenetic visual restoration.Experimental eye research · 2021Article
- AAV Induced Expression of Human Rod and Cone Opsin in Bipolar Cells of a Mouse Model of Retinal Degeneration.BioMed research international · 2021Article
Corrections and comments
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Authors and funding
19 authors at 4 institutions in 2 countries.
Charles N de LeeuwCentre for Molecular Medicine and Therapeutics at the Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada V5Z 4H4 ; Department of Medical Genetics, University of British Columbia, Vancouver, BC, Canada V5Z 4H4.
Frank M DykaDepartment of Ophthalmology, College of Medicine, University of Florida, Gainesville, FL, USA 32610.
Sanford L BoyeDepartment of Ophthalmology, College of Medicine, University of Florida, Gainesville, FL, USA 32610.
Stéphanie LapriseCentre for Molecular Medicine and Therapeutics at the Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada V5Z 4H4.
Michelle ZhouCentre for Molecular Medicine and Therapeutics at the Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada V5Z 4H4.
Alice Y ChouCentre for Molecular Medicine and Therapeutics at the Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada V5Z 4H4.
Lisa BorrettaCentre for Molecular Medicine and Therapeutics at the Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada V5Z 4H4.
Simone C McInernyCentre for Molecular Medicine and Therapeutics at the Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada V5Z 4H4.
Kathleen G BanksCentre for Molecular Medicine and Therapeutics at the Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada V5Z 4H4.
Elodie Portales-CasamarCentre for Molecular Medicine and Therapeutics at the Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada V5Z 4H4.
Magdalena I SwansonCentre for Molecular Medicine and Therapeutics at the Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada V5Z 4H4.
Cletus A D'SouzaCanada's Michael Smith Genome Sciences Centre, British Columbia Cancer Agency, Vancouver, BC, Canada V5Z 4S6.
Shannon E BoyeDepartment of Ophthalmology, College of Medicine, University of Florida, Gainesville, FL, USA 32610.
Steven J M JonesDepartment of Medical Genetics, University of British Columbia, Vancouver, BC, Canada V5Z 4H4 ; Canada's Michael Smith Genome Sciences Centre, British Columbia Cancer Agency, Vancouver, BC, Canada V5Z 4S6 ; Department of Molecular Biology and Biochemistry, Simon Fraser University, Burnaby, BC, Canada V5A 1S6.
Robert A HoltDepartment of Medical Genetics, University of British Columbia, Vancouver, BC, Canada V5Z 4H4 ; Canada's Michael Smith Genome Sciences Centre, British Columbia Cancer Agency, Vancouver, BC, Canada V5Z 4S6 ; Department of Molecular Biology and Biochemistry, Simon Fraser University, Burnaby, BC, Canada V5A 1S6 ; Department of Psychiatry, University of British Columbia, Vancouver, BC, Canada V6T 2A1.
Daniel GoldowitzCentre for Molecular Medicine and Therapeutics at the Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada V5Z 4H4 ; Department of Medical Genetics, University of British Columbia, Vancouver, BC, Canada V5Z 4H4.
William W HauswirthDepartment of Ophthalmology, College of Medicine, University of Florida, Gainesville, FL, USA 32610.
Wyeth W WassermanCentre for Molecular Medicine and Therapeutics at the Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada V5Z 4H4 ; Department of Medical Genetics, University of British Columbia, Vancouver, BC, Canada V5Z 4H4.
Elizabeth M SimpsonCentre for Molecular Medicine and Therapeutics at the Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada V5Z 4H4 ; Department of Medical Genetics, University of British Columbia, Vancouver, BC, Canada V5Z 4H4 ; Department of Psychiatry, University of British Columbia, Vancouver, BC, Canada V6T 2A1.
Child and Family Research Institute · CAUniversity of British Columbia · CAUniversity of Florida · USCanada's Michael Smith Genome Sciences Centre · CA
Funding
Vision Research CoreP30EY021721 · NEI · UNIVERSITY OF FLORIDA · PI HAUSWIRTH, WILLIAM W · 2011 to 2015
$2.8MComputational Resources for the Compilation and Distribution of Transcription FacR01GM084875 · NIGMS · UNIVERSITY OF BRITISH COLUMBIA · PI WASSERMAN, WYETH W · 2010 to 2012
$1.0MNEI NIH HHS P30 EY021721NIGMS NIH HHS R01 GM084875
Abstract
Critical for human gene therapy is the availability of small promoter tools to drive gene expression in a highly specific and reproducible manner. We tackled this challenge by developing human DNA MiniPromoters using computational biology and phylogenetic conservation. MiniPromoters were tested in mouse as single-copy knock-ins at the
Indexed as
AAVgene therapypromoters
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What OpenQuestion holds
Textmetadata
Read underepoch 390
Registered trials
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.