Targeted gene transfer to the brain via the delivery of brain-penetrating DNA nanoparticles with focused ultrasound.

[1]  A. Nairn.  Faculty of 1000 evaluation for GDNF, NGF and BDNF as therapeutic options for neurodegeneration. , 2016 .

[2]  Chih-Kuang Yeh,et al.  Focused ultrasound-induced blood-brain barrier opening for non-viral, non-invasive, and targeted gene delivery. , 2015, Journal of controlled release : official journal of the Controlled Release Society.

[3]  C. Eberhart,et al.  Highly PEGylated DNA Nanoparticles Provide Uniform and Widespread Gene Transfer in the Brain , 2015, Advanced healthcare materials.

[4]  K. Hynynen,et al.  Gene delivery to the spinal cord using MRI-guided focused ultrasound , 2015, Gene Therapy.

[5]  H. J. Baker,et al.  Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease , 2014, Gene Therapy.

[6]  E. Konofagou,et al.  Noninvasive, neuron-specific gene therapy can be facilitated by focused ultrasound and recombinant adeno-associated virus , 2014, Gene Therapy.

[7]  Elizabeth Nance,et al.  Non-invasive delivery of stealth, brain-penetrating nanoparticles across the blood-brain barrier using MRI-guided focused ultrasound. , 2014, Journal of controlled release : official journal of the Controlled Release Society.

[8]  H. Wakimoto,et al.  Oncolytic herpes simplex virus-based strategies: toward a breakthrough in glioblastoma therapy , 2014, Front. Microbiol..

[9]  S. Kügler,et al.  Pharmacologically controlled, discontinuous GDNF gene therapy restores motor function in a rat model of Parkinson's disease , 2014, Neurobiology of Disease.

[10]  Elisa E Konofagou,et al.  The Size of Blood–Brain Barrier Opening Induced by Focused Ultrasound is Dictated by the Acoustic Pressure , 2014, Journal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism.

[11]  Richard J Price,et al.  Ultrasound-activated agents comprised of 5FU-bearing nanoparticles bonded to microbubbles inhibit solid tumor growth and improve survival. , 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

[12]  Ankur Tyagi.  Focal delivery of AAV2/1-transgenes into the rat brain by localized ultrasound-induced BBB Opening , 2014, Annals of Neurosciences.

[13]  C. Agulhon,et al.  Recent molecular approaches to understanding astrocyte function in vivo , 2013, Front. Cell. Neurosci..

[14]  Kwangjoon Jeong,et al.  Molecular characterization of vulnibactin biosynthesis in Vibrio vulnificus indicates the existence of an alternative siderophore , 2013, Front. Microbiol..

[15]  Edward A. White,et al.  Multifunctional receptor-targeted nanocomplexes for the delivery of therapeutic nucleic acids to the brain. , 2013, Biomaterials.

[16]  James M. Wilson,et al.  Humoral Immune Response to AAV , 2013, Front. Immunol..

[17]  Kullervo Hynynen,et al.  Amyloid-β plaque reduction, endogenous antibody delivery and glial activation by brain-targeted, transcranial focused ultrasound , 2013, Experimental Neurology.

[18]  Max Wintermark,et al.  A pilot study of focused ultrasound thalamotomy for essential tremor. , 2013, The New England journal of medicine.

[19]  M. T. Hasan,et al.  Acat1 knockdown gene therapy decreases amyloid-β in a mouse model of Alzheimer's disease. , 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

[20]  N. Patel,et al.  GDNF, NGF and BDNF as therapeutic options for neurodegeneration. , 2013, Pharmacology & therapeutics.

[21]  M. Saarma,et al.  Gene therapy with AAV2-CDNF provides functional benefits in a rat model of Parkinson's disease , 2013, Brain and behavior.

[22]  Jie Shi,et al.  Targeted Delivery of GDNF through the Blood–Brain Barrier by MRI-Guided Focused Ultrasound , 2012, PloS one.

[23]  K. Hynynen,et al.  Enhanced delivery of gold nanoparticles with therapeutic potential into the brain using MRI-guided focused ultrasound. , 2012, Nanomedicine : nanotechnology, biology, and medicine.

[24]  Natalia Vykhodtseva,et al.  Improved anti-tumor effect of liposomal doxorubicin after targeted blood-brain barrier disruption by MRI-guided focused ultrasound in rat glioma. , 2012, Ultrasound in medicine & biology.

[25]  A. Kim,et al.  Markedly enhanced skeletal muscle transfection achieved by the ultrasound-targeted delivery of non-viral gene nanocarriers with microbubbles. , 2012, Journal of controlled release : official journal of the Controlled Release Society.

[26]  Elizabeth Nance,et al.  A Dense Poly(Ethylene Glycol) Coating Improves Penetration of Large Polymeric Nanoparticles Within Brain Tissue , 2012, Science Translational Medicine.

[27]  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.

[28]  Natalia Vykhodtseva,et al.  Temporary disruption of the blood-brain barrier by use of ultrasound and microbubbles: safety and efficacy evaluation in rhesus macaques. , 2012, Cancer research.

[29]  Yuan Cheng,et al.  Effective gene transfer into central nervous system following ultrasound-microbubbles-induced opening of the blood-brain barrier. , 2012, Ultrasound in medicine & biology.

[30]  J. Hazlehurst,et al.  Gene Therapy for Parkinson's Disease , 2012, Parkinson's disease.

[31]  S. Kügler,et al.  Efficient gene therapy for Parkinson's disease using astrocytes as hosts for localized neurotrophic factor delivery. , 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

[32]  K. Hynynen,et al.  A three-dimensional model of an ultrasound contrast agent gas bubble and its mechanical effects on microvessels , 2012, Physics in medicine and biology.

[33]  J. López-Barneo,et al.  GDNF Is Predominantly Expressed in the PV+ Neostriatal Interneuronal Ensemble in Normal Mouse and after Injury of the Nigrostriatal Pathway , 2012, The Journal of Neuroscience.

[34]  J. S. Suk,et al.  Highly compacted DNA nanoparticles with low MW PEG coatings: in vitro, ex vivo and in vivo evaluation. , 2012, Journal of controlled release : official journal of the Controlled Release Society.

[35]  Kullervo Hynynen,et al.  Targeted Delivery of Neural Stem Cells to the Brain Using MRI-Guided Focused Ultrasound to Disrupt the Blood-Brain Barrier , 2011, PloS one.

[36]  Yao-Sheng Tung,et al.  The mechanism of interaction between focused ultrasound and microbubbles in blood-brain barrier opening in mice. , 2011, The Journal of the Acoustical Society of America.

[37]  M. Tóth,et al.  In vitro and in vivo complement activation and related anaphylactic effects associated with polyethylenimine and polyethylenimine-graft-poly(ethylene glycol) block copolymers. , 2011, Biomaterials.

[38]  Richard J Price,et al.  Covalently linking poly(lactic-co-glycolic acid) nanoparticles to microbubbles before intravenous injection improves their ultrasound-targeted delivery to skeletal muscle. , 2011, Small.

[39]  M. Ehlers,et al.  Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors. , 2011, Human gene therapy.

[40]  A. Rezai,et al.  AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial , 2011, The Lancet Neurology.

[41]  A. Verkhratsky,et al.  Neuroglial Roots of Neurodegenerative Diseases? , 2011, Molecular Neurobiology.

[42]  Yunhui Liu,et al.  Mechanism of Low-Frequency Ultrasound in Opening Blood–Tumor Barrier by Tight Junction , 2011, Journal of Molecular Neuroscience.

[43]  E. Konofagou,et al.  Permeability assessment of the focused ultrasound-induced blood–brain barrier opening using dynamic contrast-enhanced MRI , 2010, Physics in medicine and biology.

[44]  R. Mandel.  CERE-110, an adeno-associated virus-based gene delivery vector expressing human nerve growth factor for the treatment of Alzheimer's disease. , 2010, Current opinion in molecular therapeutics.

[45]  J. Fyfe,et al.  Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

[46]  P. Pivirotto,et al.  Functional effects of AAV2-GDNF on the dopaminergic nigrostriatal pathway in parkinsonian rhesus monkeys. , 2009, Human gene therapy.

[47]  J. Kordower,et al.  Intrastriatal CERE-120 (AAV-Neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington's disease , 2009, Neurobiology of Disease.

[48]  In-Kyu Park,et al.  Nonviral Approaches for Neuronal Delivery of Nucleic Acids , 2007, Pharmaceutical Research.

[49]  Natalia Vykhodtseva,et al.  Targeted delivery of doxorubicin to the rat brain at therapeutic levels using MRI‐guided focused ultrasound , 2007, International journal of cancer.

[50]  L. Zhong,et al.  Self-complementary recombinant adeno-associated viral vectors: packaging capacity and the role of rep proteins in vector purity. , 2007, Human gene therapy.

[51]  Manabu Kinoshita,et al.  Noninvasive localized delivery of Herceptin to the mouse brain by MRI-guided focused ultrasound-induced blood-brain barrier disruption. , 2006, Proceedings of the National Academy of Sciences of the United States of America.

[52]  K. Hynynen,et al.  Targeted disruption of the blood–brain barrier with focused ultrasound: association with cavitation activity , 2006, Physics in medicine and biology.

[53]  Natalia Vykhodtseva,et al.  MRI-guided targeted blood-brain barrier disruption with focused ultrasound: histological findings in rabbits. , 2005, Ultrasound in medicine & biology.

[54]  Ferenc A. Jolesz,et al.  Local and reversible blood–brain barrier disruption by noninvasive focused ultrasound at frequencies suitable for trans-skull sonications , 2005, NeuroImage.

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

[56]  R. Mandel,et al.  Circulating Anti-Wild-Type Adeno-Associated Virus Type 2 (AAV2) Antibodies Inhibit Recombinant AAV2 (rAAV2)-Mediated, but Not rAAV5-Mediated, Gene Transfer in the Brain , 2004, Journal of Virology.

[57]  Theresa A. Storm,et al.  Rapid Uncoating of Vector Genomes Is the Key toEfficient Liver Transduction with Pseudotyped Adeno-Associated VirusVectors , 2004, Journal of Virology.

[58]  G. Chadi,et al.  ASTROGLIAL AND MICROGLIAL ACTIVATION IN THE WISTAR RAT VENTRAL TEGMENTAL AREA AFTER A SINGLE STRIATAL INJECTION OF 6-HYDROXYDOPAMINE , 2004, The International journal of neuroscience.

[59]  Clive J Roberts,et al.  Polyethylenimine-graft-poly(ethylene glycol) copolymers: influence of copolymer block structure on DNA complexation and biological activities as gene delivery system. , 2002, Bioconjugate chemistry.

[60]  K. Hynynen,et al.  Noninvasive MR imaging-guided focal opening of the blood-brain barrier in rabbits. , 2001, Radiology.

[61]  L. Barrett,et al.  Gene therapy for central nervous system repair. , 2001, Current opinion in molecular therapeutics.

[62]  A. Mikos,et al.  Poly(ethylenimine) and its role in gene delivery. , 1999, Journal of controlled release : official journal of the Controlled Release Society.

[63]  E. Neuwelt,et al.  Outwitting the blood-brain barrier for therapeutic purposes: osmotic opening and other means. , 1998, Neurosurgery.

[64]  M. Kay,et al.  The kinetics of rAAV integration in the liver , 1998, Nature Genetics.

[65]  P. Kornblith.  Outwitting the Blood-Brain Barrier for Therapeutic Purposes: Osmotic Opening and Other Means , 1998 .

[66]  G. Barsh,et al.  Mitochondrial transcription factor A is necessary for mtDNA maintance and embryogenesis in mice , 1998, Nature Genetics.

[67]  P F Morrison,et al.  Convection-enhanced delivery of macromolecules in the brain. , 1994, Proceedings of the National Academy of Sciences of the United States of America.

[68]  J. O'Callaghan,et al.  The concentration of glial fibrillary acidic protein increases with age in the mouse and rat brain , 1991, Neurobiology of Aging.

[69]  S. Gray,et al.  Clinical applications involving CNS gene transfer. , 2014, Advances in genetics.

[70]  W. J. Elias,et al.  Neuromodulation for movement disorders. , 2014, Neurosurgery clinics of North America.

[71]  W. Pardridge.  The blood-brain barrier: Bottleneck in brain drug development , 2005, NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics.

[72]  A. Aigner,et al.  PEG grafting of polyethylenimine (PEI) exerts different effects on DNA transfection and siRNA-induced gene targeting efficacy. , 2008, Journal of drug targeting.

[73]  W. Pardridge,et al.  Blood-brain barrier delivery. , 2007, Drug discovery today.

[74]  D. Deleu,et al.  Clinical Pharmacokinetic and Pharmacodynamic Properties of Drugs Used in the Treatment of Parkinson’s Disease , 2002, Clinical pharmacokinetics.

[75]  D. Ovchinnikov,et al.  Limb and kidney defects in Lmx1b mutant mice suggest an involvement of LMX1B in human nail patella syndrome , 1998, Nature Genetics.