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)
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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)
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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
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.
Published in Nature communications (12.07.2021)
Published in Nature communications (12.07.2021)
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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
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)
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Trial of magnetic resonance–guided putaminal gene therapy for advanced Parkinson's disease
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.
Published in Movement disorders (01.07.2019)
Published in Movement disorders (01.07.2019)
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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
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)
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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 (01.10.2018)
Published in Nature reviews. Drug discovery (01.10.2018)
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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)
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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)
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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)
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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
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.
Published in Stem cells (Dayton, Ohio) (01.07.2018)
Published in Stem cells (Dayton, Ohio) (01.07.2018)
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Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial
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
Published in Neurology (04.01.2022)
Published in Neurology (04.01.2022)
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Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates
Samaranch, Lluis, Salegio, Ernesto A, San Sebastian, Waldy, Kells, Adrian P, Bringas, John R, Forsayeth, John, Bankiewicz, Krystof S
Published in Human gene therapy (01.05.2013)
Published in Human gene therapy (01.05.2013)
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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
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.
Published in Molecular therapy (07.12.2022)
Published in Molecular therapy (07.12.2022)
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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)
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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)
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Infuse-as-you-go convective delivery to enhance coverage of elongated brain targets: technical note
Sudhakar, Vivek, Naidoo, Jerusha, Samaranch, Lluis, Bringas, John R, Lonser, Russell R, Fiandaca, Massimo S, Bankiewicz, Krystof S
Published in Journal of neurosurgery (01.08.2020)
Published in Journal of neurosurgery (01.08.2020)
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