ArticleScience (New York, N.Y.)2024
An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery.
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Article in Science (New York, N.Y.), 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 118 papers.
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118citing papers in PubMed
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Who cites it
118 citing papers in PubMed.
- AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial.The New England journal of medicine · 2026Trial
- Benchmarking the next generation of brain-penetrant AAV capsids.Molecular therapy. Advances · 2026Article
- Controllable gene delivery via masked adeno-associated viral vectors.Nature materials · 2026Article
- AAV-mediated gene therapy demonstrates phenotypic rescue in a mouse model of Cockayne syndrome.The Journal of clinical investigation · 2026Article
- i.c.v. delivery of AAV9-synapsin-promoted caveolin-1 attenuates neuromuscular deficits and neuromuscular degeneration in hSOD1Molecular therapy. Advances · 2026Article
- Antisense oligonucleotide treatment following viral delivery of artificial SOD1-targeting miRNA shows improved efficacy in SOD1-G93A mice.Molecular therapy. Advances · 2026Article
- Article
- Phenotypic screening for small molecules that lower PrP in cultured cells.Molecular and cellular neurosciences · 2026Article
- Cellular and signalling mechanisms that regulate the blood-brain barrier.Nature reviews. Molecular cell biology · 2026Review
- Breaking Biological Barriers: Engineering Nanocarriers for Efficient Drug and Gene Delivery through Nano-Bio Interfaces and Biophysical Design.Applied physics reviews · 2026Article
- CA-IV-directed small-molecule shuttle enables targeted brain delivery of biologics.Nature chemical biology · 2026Article
- Shifting AAV9 tropism by binding moiety insertion enhances transduction of tumor cell lines and human glioblastoma explants.Scientific reports · 2026Article
- Unraveling competitive protein interactions: A critical step toward improved production and therapeutics.Journal of advanced research · 2026Review
- Systematic Design and Evaluation of Nasal Drug Delivery for Central Nervous System Disease from Experimental to Clinical Application.Pharmaceutics · 2026Review
- Epigenetic editing approaches maturity: AI-driven precision design, delivery innovation, and the road to clinical translation.Clinical epigenetics · 2026Review
- Efficient targeting of human glial progenitor cells in vivo with engineered AAV vectors and glymphatic delivery.Nature biotechnology · 2026Article
- An AAV vector for glial cells.Nature biotechnology · 2026Article
- Safety considerations of gene-based therapies for Alzheimer's disease.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2026Review
- Structural basis of liver de-targeting and neuronal tropism of CNS-targeted AAV capsids.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- From Gene to Hope: Rett Syndrome and the Rise of Molecular Therapies.Molecular diagnosis & therapy · 2026Review
58 more citing papers are in PubMed but not listed here.
Corrections and comments
- Update of
Authors and funding
24 authors.
Qin Huang *Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0000-0002-7170-7455
Ken Y Chan *Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0000-0002-8853-5186
Jason WuStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0000-0003-3036-9928
Nuria R Botticello-RomeroStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0009-0009-5866-177X
Qingxia ZhengStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0009-0009-9138-6853
Shan LouStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0000-0002-1638-9917
Casey KeyesStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0000-0001-8763-0221
Alexander SvanbergssonStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0000-0003-4435-7653
Jencilin JohnstonStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.
Allan MillsStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0000-0003-3056-7764
Chin-Yen LinStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0009-0004-3948-2307
Pamela P BrauerStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.
Gabrielle ClouseStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.
Simon PacouretStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.
John W HarveyStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.
Thomas BeddowStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.
Jenna K HurleyStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0009-0005-4477-853X
Isabelle G TobeyStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0000-0001-5428-3809
Megan PowellStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.
Albert T ChenStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0000-0002-5387-0208
Andrew J BarryStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0000-0002-4667-679X
Fatma-Elzahraa EidStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.
Yujia A ChanStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0000-0002-0731-637X
Benjamin E DevermanStanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142 USA.ORCID 0000-0002-6223-9303
Funding
Development and validation of AAV vectors to manipulate specific neuronal subtypes and circuits involved in epilepsy and psychiatric disorders across mammalian species.UG3MH120096 · NIMH · BROAD INSTITUTE, INC. · PI DEVERMAN, BENJAMIN E, FISHELL, GORDON J · 2019 to 2021
$6.7MDevelopment and validation of AAV vectors to manipulate specific neuronal subtypes and circuits involved in epilepsy and psychiatric disorders across mammalian species.UH3MH120096 · NIMH · BROAD INSTITUTE, INC. · PI DEVERMAN, BENJAMIN E, FISHELL, GORDON J · 2023 to 2024
$4.7MNovel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous SystemUH3NS111689 · NINDS · BROAD INSTITUTE, INC. · PI DEVERMAN, BENJAMIN E · 2021 to 2022
$2.7MNovel AAVs engineered for efficient and noninvasive cross-species gene editing throughout the central nervous systemUG3NS111689 · NINDS · BROAD INSTITUTE, INC. · PI DEVERMAN, BENJAMIN E · 2018 to 2020
$2.5MNIMH NIH HHS UG3 MH120096NIMH NIH HHS UH3 MH120096NINDS NIH HHS UG3 NS111689NINDS NIH HHS UH3 NS111689
Abstract
Developing vehicles that efficiently deliver genes throughout the human central nervous system (CNS) will broaden the range of treatable genetic diseases. We engineered an adeno-associated virus (AAV) capsid, BI-hTFR1, that binds human transferrin receptor (TfR1), a protein expressed on the blood-brain barrier. BI-hTFR1 was actively transported across human brain endothelial cells and, relative to AAV9, provided 40 to 50 times greater reporter expression in the CNS of human
Indexed as
Antigens, CDBrainCapsidGenetic VectorsGene Transfer TechniquesGlucosylceramidaseReceptors, TransferrinAnimalsBlood-Brain BarrierCapsid ProteinsDependovirusEndothelial CellsGaucher DiseaseGene Knock-In TechniquesGenetic TherapyHumansAntigens, CDCapsid ProteinsCD71 antigenGBA protein, humanGlucosylceramidaseReceptors, Transferrin
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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.