Gene therapy for neurological disorders: progress and prospects
Deverman, Benjamin E, Ravina, Bernard M, Bankiewicz, Krystof S, Paul, Steven M, Sah, Dinah W Y
Published in Nature reviews. Drug discovery (01.09.2018)
Published in Nature reviews. Drug discovery (01.09.2018)
Get full text
Journal Article
Magnetic resonance imaging–guided phase 1 trial of putaminal AADC gene therapy for Parkinson's disease
Christine, Chadwick W., Bankiewicz, Krystof S., Van Laar, Amber D., Richardson, R. Mark, Ravina, Bernard, Kells, Adrian P., Boot, Brendon, Martin, Alastair J., Nutt, John, Thompson, Marin E., Larson, Paul S.
Published in Annals of neurology (01.05.2019)
Published in Annals of neurology (01.05.2019)
Get full text
Journal Article
AAV9-mediated Expression of a Non-self Protein in Nonhuman Primate Central Nervous System Triggers Widespread Neuroinflammation Driven by Antigen-presenting Cell Transduction
Samaranch, Lluis, Sebastian, Waldy San, Kells, Adrian P, Salegio, Ernesto A, Heller, Gregory, Bringas, John R, Pivirotto, Philip, DeArmond, Stephen, Forsayeth, John, Bankiewicz, Krystof S
Published in Molecular therapy (01.02.2014)
Published in Molecular therapy (01.02.2014)
Get full text
Journal Article
Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates
Samaranch, Lluis, Salegio, Ernesto A, San Sebastian, Waldy, Kells, Adrian P, Foust, Kevin D, Bringas, John R, Lamarre, Clementine, Forsayeth, John, Kaspar, Brian K, Bankiewicz, Krystof S
Published in Human gene therapy (01.04.2012)
Published in Human gene therapy (01.04.2012)
Get more information
Journal Article
Cerebral Infusion of AAV9 Vector-encoding Non-self Proteins Can Elicit Cell-mediated Immune Responses
Ciesielska, Agnieszka, Hadaczek, Piotr, Mittermeyer, Gabriele, Zhou, Shangzhen, Wright, J Fraser, Bankiewicz, Krystof S, Forsayeth, John
Published in Molecular therapy (01.01.2013)
Published in Molecular therapy (01.01.2013)
Get full text
Journal Article
Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease
Mittermeyer, Gabriele, Christine, Chadwick W, Rosenbluth, Kathryn H, Baker, Suzanne L, Starr, Philip, Larson, Paul, Kaplan, Paul L, Forsayeth, John, Aminoff, Michael J, Bankiewicz, Krystof S
Published in Human gene therapy (01.04.2012)
Published in Human gene therapy (01.04.2012)
Get more information
Journal Article
Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS
Naidoo, Jerusha, Stanek, Lisa M., Ohno, Kousaku, Trewman, Savanah, Samaranch, Lluis, Hadaczek, Piotr, O’Riordan, Catherine, Sullivan, Jennifer, San Sebastian, Waldy, Bringas, John R., Snieckus, Christopher, Mahmoodi, Amin, Mahmoodi, Amir, Forsayeth, John, Bankiewicz, Krystof S., Shihabuddin, Lamya S.
Published in Molecular therapy (03.10.2018)
Published in Molecular therapy (03.10.2018)
Get full text
Journal Article
Adeno-associated viral vector serotype 9-based gene therapy for Niemann-Pick disease type A
Samaranch, Lluis, Pérez-Cañamás, Azucena, Soto-Huelin, Beatriz, Sudhakar, Vivek, Jurado-Arjona, Jerónimo, Hadaczek, Piotr, Ávila, Jesús, Bringas, John R, Casas, Josefina, Chen, Haifeng, He, Xingxuan, Schuchman, Edward H, Cheng, Seng H, Forsayeth, John, Bankiewicz, Krystof S, Ledesma, María Dolores
Published in Science translational medicine (21.08.2019)
Published in Science translational medicine (21.08.2019)
Get more information
Journal Article
Development of a novel frameless skull-mounted ball-joint guide array for use in image-guided neurosurgery
Sudhakar, Vivek, Mahmoodi, Amin, Bringas, John R, Naidoo, Jerusha, Kells, Adrian, Samaranch, Lluis, Fiandaca, Massimo S, Bankiewicz, Krystof S
Published in Journal of neurosurgery (01.02.2020)
Published in Journal of neurosurgery (01.02.2020)
Get more information
Journal Article
Depletion of AADC activity in caudate nucleus and putamen of Parkinson's disease patients; implications for ongoing AAV2-AADC gene therapy trial
Ciesielska, Agnieszka, Samaranch, Lluis, San Sebastian, Waldy, Dickson, Dennis W, Goldman, Samuel, Forsayeth, John, Bankiewicz, Krystof S
Published in PloS one (06.02.2017)
Published in PloS one (06.02.2017)
Get full text
Journal Article
Interventional MRI-guided Putaminal Delivery of AAV2-GDNF for a Planned Clinical Trial in Parkinson's Disease
Richardson, R Mark, Kells, Adrian P, Rosenbluth, Kathryn H, Salegio, Ernesto Aguilar, Fiandaca, Massimo S, Larson, Paul S, Starr, Philip A, Martin, Alastair J, Lonser, Russell R, Federoff, Howard J, Forsayeth, John R, Bankiewicz, Krystof S
Published in Molecular therapy (01.06.2011)
Published in Molecular therapy (01.06.2011)
Get full text
Journal Article
Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy
Passini, Marco A, Bu, Jie, Richards, Amy M, Treleaven, Christopher M, Sullivan, Jennifer A, O'Riordan, Catherine R, Scaria, Abraham, Kells, Adrian P, Samaranch, Lluis, San Sebastian, Waldy, Federici, Thais, Fiandaca, Massimo S, Boulis, Nicholas M, Bankiewicz, Krystof S, Shihabuddin, Lamya S, Cheng, Seng H
Published in Human gene therapy (01.07.2014)
Published in Human gene therapy (01.07.2014)
Get more information
Journal Article
Efficient gene therapy-based method for the delivery of therapeutics to primate cortex
Kells, Adrian P, Hadaczek, Piotr, Yin, Dali, Bringas, John, Varenika, Vanja, Forsayeth, John, Bankiewicz, Krystof S
Published in Proceedings of the National Academy of Sciences - PNAS (17.02.2009)
Published in Proceedings of the National Academy of Sciences - PNAS (17.02.2009)
Get full text
Journal Article
Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates
Ohno, Kousaku, Samaranch, Lluis, Hadaczek, Piotr, Bringas, John R., Allen, Philip C., Sudhakar, Vivek, Stockinger, Diane E., Snieckus, Christopher, Campagna, Michael V., San Sebastian, Waldy, Naidoo, Jerusha, Chen, Haifeng, Forsayeth, John, Salegio, Ernesto A., Hwa, Granger G.C., Bankiewicz, Krystof S.
Published in Molecular therapy. Methods & clinical development (14.06.2019)
Published in Molecular therapy. Methods & clinical development (14.06.2019)
Get full text
Journal Article
Functional effects of AAV2-GDNF on the dopaminergic nigrostriatal pathway in parkinsonian rhesus monkeys
Eberling, Jamie L, Kells, Adrian P, Pivirotto, Philip, Beyer, Janine, Bringas, John, Federoff, Howard J, Forsayeth, John, Bankiewicz, Krystof S
Published in Human gene therapy (01.05.2009)
Published in Human gene therapy (01.05.2009)
Get more information
Journal Article
Clinically relevant effects of convection-enhanced delivery of AAV2-GDNF on the dopaminergic nigrostriatal pathway in aged rhesus monkeys
Johnston, Louisa C, Eberling, Jamie, Pivirotto, Philip, Hadaczek, Piotr, Federoff, Howard J, Forsayeth, John, Bankiewicz, Krystof S
Published in Human gene therapy (01.05.2009)
Published in Human gene therapy (01.05.2009)
Get more information
Journal Article
Distribution of nanoparticles throughout the cerebral cortex of rodents and non-human primates: Implications for gene and drug therapy
Salegio, Ernesto A, Streeter, Hillary, Dube, Nikhil, Hadaczek, Piotr, Samaranch, Lluis, Kells, Adrian P, San Sebastian, Waldy, Zhai, Yuying, Bringas, John, Xu, Ting, Forsayeth, John, Bankiewicz, Krystof S
Published in Frontiers in neuroanatomy (17.03.2014)
Published in Frontiers in neuroanatomy (17.03.2014)
Get full text
Journal Article
Novel nanoliposomal CPT-11 infused by convection-enhanced delivery in intracranial tumors : Pharmacology and efficacy
NOBLE, Charles O, KRAUZE, Michal T, DRUMMOND, Daryl C, YAMASHITA, Yoji, SAITO, Ryuta, BERGER, Mitchel S, KIRPOTIN, Dmitri B, BANKIEWICZ, Krystof S, PARK, John W
Published in Cancer research (Chicago, Ill.) (01.03.2006)
Published in Cancer research (Chicago, Ill.) (01.03.2006)
Get full text
Journal Article
Convection-enhanced delivery of nanoliposomal CPT-11 (irinotecan) and PEGylated liposomal doxorubicin (Doxil) in rodent intracranial brain tumor xenografts
Krauze, Michal T, Noble, Charles O, Kawaguchi, Tomohiro, Drummond, Daryl, Kirpotin, Dmitri B, Yamashita, Yoji, Kullberg, Erika, Forsayeth, John, Park, John W, Bankiewicz, Krystof S
Published in Neuro-oncology (Charlottesville, Va.) (01.10.2007)
Published in Neuro-oncology (Charlottesville, Va.) (01.10.2007)
Get full text
Journal Article