Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Systemic Administration of AAV9-G266A with Focused Ultrasound Enables Robust Brain Transduction with Minimal Liver Transduction.

Human gene therapy - 1 Mar 2026

Nelson Andrew T, Smith Aidan, Curley Colleen T, Kelly Brian, Ganesh Ahil N, Peterson Drew, Fabyanic Emily, Kostuk Eric, Weber Thomas, Engel Esteban A

Abstract excerpt

Systemic delivery of adeno-associated viral (AAV) vectors is a promising approach for brain gene therapy, particularly in combination with emerging techniques such as focused ultrasound (FUS), which can transiently and noninvasively open the blood-brain barrier to facilitate delivery of AAVs to the brain. However, off-target vector accumulation, particularly in the liver, remains a significant safety concern. To...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Brain
Share to Capsid Proteins

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Dependovirus

Share to Gene Transfer Techniques

Share to Genetic Therapy

Share to Genetic Vectors

Share to Transduction, Genetic

Source

Identifiers

PMID: 41656626
DOI: 10.1177/10430342251411044

Genes

FUS

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Systemic Administration of AAV9-G266A with Focused Ultrasound Enables Robust Brain Transduction with Minimal Liver Transduction.PMID 41656626Read ↗

Engineering viral vectors for acoustically targeted gene deliverySemantic proximity Read ↗

Engineering Viral Vectors for Acoustically Targeted Gene DeliverySemantic proximity Read ↗

Novel Engineered AAV Variants Demonstrate Superior Blood-Brain Barrier Penetration and Safety in Non-Human PrimatesSemantic proximity Read ↗

PET imaging of focused-ultrasound enhanced delivery of AAVs into the murine brainSemantic proximity Read ↗

New AAV9 engineered variants with enhanced neurotropism and reduced liver off-targeting in mice and marmosetsSemantic proximity Read ↗

Routes of administration for adeno-associated viruses carrying gene therapies for brain diseasesSemantic proximity Read ↗

LRP6-Guided Engineering of AAV9 Variants with Enhanced Blood-Brain Barrier Penetration and Reduced Liver Tropism in Non-Human PrimatesSemantic proximity Read ↗

Portable transcranial therapeutic ultrasound enhances targeted gene delivery for Parkinson’s disease: from rodent models to non-human primatesSemantic proximity Read ↗

Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primatesSemantic proximity Read ↗

Rational Design of AAV-rh74, AAV3B, and AAV8 with Limited Liver TargetingSemantic proximity Read ↗

Systemic gene delivery to the central nervous system using Adeno-associated virusSemantic proximity Read ↗

Adeno-associated virus (AAV) capsid engineering in liver-directed gene therapySemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.