Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression

[1]  K. Hynynen,et al.  Efficacy of gene delivery to the brain using AAV and ultrasound depends on serotypes and brain areas. , 2022, Journal of controlled release : official journal of the Controlled Release Society.

[2]  K. Hynynen,et al.  The engineered AAV2-HBKO promotes non-invasive gene delivery to large brain regions beyond ultrasound targeted sites , 2022, Molecular therapy. Methods & clinical development.

[3]  K. Hynynen,et al.  Microbubble drug conjugate and focused ultrasound blood brain barrier delivery of AAV-2 SIRT-3 , 2022, Drug delivery.

[4]  E. Simpson,et al.  Transgene distribution and immune response following focused ultrasound delivery to the brain of rAAV9 and PHP.B in a mouse model of Alzheimer disease , 2021, Molecular Therapy - Methods & Clinical Development.

[5]  A. Philippidis,et al.  Fourth Boy Dies in Clinical Trial of Astellas' AT132. , 2021, Human gene therapy.

[6]  Rachelle A. Farkas,et al.  Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells , 2021, Gene Therapy.

[7]  S. Kügler,et al.  Systemic AAV6-synapsin-GFP administration results in lower liver biodistribution, compared to AAV1&2 and AAV9, with neuronal expression following ultrasound-mediated brain delivery , 2021, Scientific Reports.

[8]  Takeshi Nakajima,et al.  Intra-cisterna magna delivery of an AAV vector with the GLUT1 promoter in a pig recapitulates the physiological expression of SLC2A1 , 2020, Gene Therapy.

[9]  Michael J. Castle,et al.  Postmortem Analysis in a Clinical Trial of AAV2-NGF Gene Therapy for Alzheimer's Disease Identifies a Need for Improved Vector Delivery. , 2020, Human gene therapy.

[10]  G. Miller,et al.  Sonoselective transfection of cerebral vasculature without blood–brain barrier disruption , 2020, Proceedings of the National Academy of Sciences.

[11]  Steven J. M. Jones,et al.  Targeted CNS delivery using human MiniPromoters and demonstrated compatibility with adeno-associated viral vectors , 2014, Molecular therapy. Methods & clinical development.

[12]  K. Hynynen,et al.  Targeted delivery of self-complementary adeno-associated virus serotype 9 to the brain, using magnetic resonance imaging-guided focused ultrasound. , 2012, Human gene therapy.

[13]  S. Meairs,et al.  Elucidating the mechanisms behind sonoporation with adeno-associated virus-loaded microbubbles. , 2011, Molecular pharmaceutics.

[14]  Kullervo Hynynen,et al.  Effect of focused ultrasound applied with an ultrasound contrast agent on the tight junctional integrity of the brain microvascular endothelium. , 2008, Ultrasound in medicine & biology.

[15]  Kullervo Hynynen,et al.  Brain arterioles show more active vesicular transport of blood-borne tracer molecules than capillaries and venules after focused ultrasound-evoked opening of the blood-brain barrier. , 2006, Ultrasound in medicine & biology.

[16]  K. Hynynen,et al.  Cellular mechanisms of the blood-brain barrier opening induced by ultrasound in presence of microbubbles. , 2004, Ultrasound in medicine & biology.