Search Results - "Bankiewicz, Krystof S."

Gene therapy for neurological disorders: progress and prospects by Deverman, Benjamin E., Ravina, Bernard M., Bankiewicz, Krystof S., Paul, Steven M., Sah, Dinah W. Y.

“…The potential of adeno-associated viral (AAV)-mediated gene therapy for neurological disorders is rapidly emerging. Evidence of clinical efficacy and safety,…”
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Magnetic resonance imaging–guided phase 1 trial of putaminal AADC gene therapy for Parkinson's disease by 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.

“…Objective To understand the safety, putaminal coverage, and enzyme expression of adeno‐associated viral vector serotype‐2 encoding the complementary DNA for…”
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Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons by Pearson, Toni S., Gupta, Nalin, San Sebastian, Waldy, Imamura-Ching, Jill, Viehoever, Amy, Grijalvo-Perez, Ana, Fay, Alex J., Seth, Neha, Lundy, Shannon M., Seo, Youngho, Pampaloni, Miguel, Hyland, Keith, Smith, Erin, de Oliveira Barbosa, Gardenia, Heathcock, Jill C., Minnema, Amy, Lonser, Russell, Elder, J. Bradley, Leonard, Jeffrey, Larson, Paul, Bankiewicz, Krystof S.

“…Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder characterized by deficient synthesis of dopamine and serotonin. It presents in…”
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AAV9-mediated Expression of a Non-self Protein in Nonhuman Primate Central Nervous System Triggers Widespread Neuroinflammation Driven by Antigen-presenting Cell Transduction by 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

“…Many studies have demonstrated that adeno-associated virus serotype 9 (AAV9) transduces astrocytes and neurons when infused into rat or nonhuman primate (NHP)…”
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Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates by 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

“…Widespread distribution of gene products at clinically relevant levels throughout the CNS has been challenging. Adeno-associated virus type 9 (AAV9) vector has…”
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Cerebral Infusion of AAV9 Vector-encoding Non-self Proteins Can Elicit Cell-mediated Immune Responses by Ciesielska, Agnieszka, Hadaczek, Piotr, Mittermeyer, Gabriele, Zhou, Shangzhen, Wright, J Fraser, Bankiewicz, Krystof S, Forsayeth, John

“…There is considerable interest in the use of adeno-associated virus serotype 9 (AAV9) for neurological gene therapy partly because of its ability to cross the…”
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Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease by 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

“…We report the results of a long-term follow-up of subjects in a phase 1 study of AAV2-hAADC (adeno-associated virus type 2-human aromatic L-amino acid…”
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Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS by 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.

“…The present study was designed to characterize transduction of non-human primate brain and spinal cord with a modified adeno-associated virus serotype 2,…”
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Development of a novel frameless skull-mounted ball-joint guide array for use in image-guided neurosurgery by Sudhakar, Vivek, Mahmoodi, Amin, Bringas, John R, Naidoo, Jerusha, Kells, Adrian, Samaranch, Lluis, Fiandaca, Massimo S, Bankiewicz, Krystof S

“…Successful convection-enhanced delivery of therapeutic agents to subcortical brain structures requires accurate cannula placement. Stereotactic guiding devices…”
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Glial-derived neurotrophic factor gene transfer for Parkinson's disease: Anterograde distribution of AAV2 vectors in the primate brain by Kells, Adrian P, Forsayeth, John, Bankiewicz, Krystof S

“…Abstract Delivery of neurotrophic factors to treat neurodegenerative diseases has not been efficacious in clinical trials despite their known potency for…”
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Depletion of AADC activity in caudate nucleus and putamen of Parkinson's disease patients; implications for ongoing AAV2-AADC gene therapy trial by Ciesielska, Agnieszka, Samaranch, Lluis, San Sebastian, Waldy, Dickson, Dennis W, Goldman, Samuel, Forsayeth, John, Bankiewicz, Krystof S

“…In Parkinson's disease (PD), aromatic L-amino acid decarboxylase (AADC) is the rate-limiting enzyme in the conversion of L-DOPA (Sinemet) to dopamine (DA)…”
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Interventional MRI-guided Putaminal Delivery of AAV2-GDNF for a Planned Clinical Trial in Parkinson's Disease by 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

“…Clinical trials involving direct infusion of neurotrophic therapies for Parkinson's disease (PD) have suffered from poor coverage of the putamen. The planned…”
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Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy by 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

“…Spinal muscular atrophy (SMA) is a neuromuscular disease caused by mutations in survival motor neuron 1 (SMN1). Previously, we showed that central nervous…”
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Efficient gene therapy-based method for the delivery of therapeutics to primate cortex by Kells, Adrian P, Hadaczek, Piotr, Yin, Dali, Bringas, John, Varenika, Vanja, Forsayeth, John, Bankiewicz, Krystof S

“…Transduction of the primate cortex with adeno-associated virus (AAV)-based gene therapy vectors has been challenging, because of the large size of the cortex…”
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Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates by 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.

“…Here we evaluated the utility of MRI to monitor intrathecal infusions in nonhuman primates. Adeno-associated virus (AAV) spiked with gadoteridol, a…”
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Functional effects of AAV2-GDNF on the dopaminergic nigrostriatal pathway in parkinsonian rhesus monkeys by Eberling, Jamie L, Kells, Adrian P, Pivirotto, Philip, Beyer, Janine, Bringas, John, Federoff, Howard J, Forsayeth, John, Bankiewicz, Krystof S

“…We investigated the safety and neuroregenerative potential of an adeno-associated virus (AAV2) containing human glial cell line-derived neurotrophic factor…”
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Clinically relevant effects of convection-enhanced delivery of AAV2-GDNF on the dopaminergic nigrostriatal pathway in aged rhesus monkeys by Johnston, Louisa C, Eberling, Jamie, Pivirotto, Philip, Hadaczek, Piotr, Federoff, Howard J, Forsayeth, John, Bankiewicz, Krystof S

“…Growth factor therapy for Parkinson's disease offers the prospect of restoration of dopaminergic innervation and/or prevention of neurodegeneration. Safety and…”
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Distribution of nanoparticles throughout the cerebral cortex of rodents and non-human primates: Implications for gene and drug therapy by 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

“…When nanoparticles/proteins are infused into the brain, they are often transported to distal sites in a manner that is dependent both on the characteristics of…”
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Novel nanoliposomal CPT-11 infused by convection-enhanced delivery in intracranial tumors : Pharmacology and efficacy by 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

“…We hypothesized that combining convection-enhanced delivery (CED) with a novel, highly stable nanoparticle/liposome containing CPT-11 (nanoliposomal CPT-11)…”
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Convection-enhanced delivery of nanoliposomal CPT-11 (irinotecan) and PEGylated liposomal doxorubicin (Doxil) in rodent intracranial brain tumor xenografts by 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

“…We have previously shown that convection-enhanced delivery (CED) of highly stable nanoparticle/liposome agents encapsulating chemotherapeutic drugs is…”
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