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

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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 von 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.

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Magnetic resonance imaging–guided phase 1 trial of putaminal AADC gene therapy for Parkinson's disease von 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.

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Direct convective delivery of adeno-associated virus gene therapy for treatment of neurological disorders von Lonser, Russell R, Akhter, Asad S, Zabek, Mirosław, Elder, J Bradley, Bankiewicz, Krystof S

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Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates von 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

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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 von 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

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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 von Ciesielska, Agnieszka, Samaranch, Lluis, San Sebastian, Waldy, Dickson, Dennis W, Goldman, Samuel, Forsayeth, John, Bankiewicz, Krystof S

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Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease von 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

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Cerebral Infusion of AAV9 Vector-encoding Non-self Proteins Can Elicit Cell-mediated Immune Responses von Ciesielska, Agnieszka, Hadaczek, Piotr, Mittermeyer, Gabriele, Zhou, Shangzhen, Wright, J Fraser, Bankiewicz, Krystof S, Forsayeth, John

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Trial of magnetic resonance–guided putaminal gene therapy for advanced Parkinson's disease von Heiss, John D., Lungu, Codrin, Hammoud, Dima A., Herscovitch, Peter, Ehrlich, Debra J., Argersinger, Davis P., Sinharay, Sanhita, Scott, Gretchen, Wu, Tianxia, Federoff, Howard J., Zaghloul, Kareem A., Hallett, Mark, Lonser, Russell R., Bankiewicz, Krystof S.

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

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AAV viral vector delivery to the brain by shape-conforming MR-guided infusions von Bankiewicz, Krystof S., Sudhakar, Vivek, Samaranch, Lluis, San Sebastian, Waldy, Bringas, John, Forsayeth, John

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Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS von 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.

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Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial von Christine, Chadwick W., Richardson, R. Mark, Van Laar, Amber D., Thompson, Marin E., Fine, Elisabeth M., Khwaja, Omar S., Li, Chunming, Liang, Grace S., Meier, Andreas, Roberts, Eiry W., Pfau, Madeline L., Rodman, Josh R., Bankiewicz, Krystof S., Larson, Paul S.

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Transplantation of Neural Progenitor Cells Expressing Glial Cell Line‐Derived Neurotrophic Factor into the Motor Cortex as a Strategy to Treat Amyotrophic Lateral Sclerosis von Thomsen, Gretchen M., Avalos, Pablo, Ma, Annie A., Alkaslasi, Mor, Cho, Noell, Wyss, Livia, Vit, Jean‐Philippe, Godoy, Marlesa, Suezaki, Patrick, Shelest, Oksana, Bankiewicz, Krystof S., Svendsen, Clive N.

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Long-term safety of MRI-guided administration of AAV2-GDNF and gadoteridol in the putamen of individuals with Parkinson’s disease von Rocco, Matthew T., Akhter, Asad S., Ehrlich, Debra J., Scott, Gretchen C., Lungu, Codrin, Munjal, Vikas, Aquino, Anthony, Lonser, Russell R., Fiandaca, Massimo S., Hallett, Mark, Heiss, John D., Bankiewicz, Krystof S.

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Comprehensive in vivo mapping of the human basal ganglia and thalamic connectome in individuals using 7T MRI von Lenglet, Christophe, Abosch, Aviva, Yacoub, Essa, De Martino, Federico, Sapiro, Guillermo, Harel, Noam

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Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates von Samaranch, Lluis, Salegio, Ernesto A, San Sebastian, Waldy, Kells, Adrian P, Bringas, John R, Forsayeth, John, Bankiewicz, Krystof S

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Development of a novel frameless skull-mounted ball-joint guide array for use in image-guided neurosurgery von Sudhakar, Vivek, Mahmoodi, Amin, Bringas, John R, Naidoo, Jerusha, Kells, Adrian, Samaranch, Lluis, Fiandaca, Massimo S, Bankiewicz, Krystof S

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Infuse-as-you-go convective delivery to enhance coverage of elongated brain targets: technical note von Sudhakar, Vivek, Naidoo, Jerusha, Samaranch, Lluis, Bringas, John R, Lonser, Russell R, Fiandaca, Massimo S, Bankiewicz, Krystof S

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