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Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome
Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome
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Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

Boztug, Kaan ; Schmidt, Manfred ; Schwarzer, Adrian ; Banerjee, Pinaki P ; Díez, Inés Avedillo ; Dewey, Ricardo A ; Böhm, Marie ; Nowrouzi, Ali ; Ball, Claudia R ; Glimm, Hanno ; Naundorf, Sonja ; Kühlcke, Klaus ; Blasczyk, Rainer ; Kondratenko, Irina ; Maródi, László ; Orange, Jordan S ; von Kalle, Christof ; Klein, Christoph

The New England journal of medicine, 2010-11, Vol.363 (20), p.1918-1927 [Periódico revisado por pares]

Waltham, MA: Massachusetts Medical Society

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2
Limbal Stem-Cell Therapy and Long-Term Corneal Regeneration
Limbal Stem-Cell Therapy and Long-Term Corneal Regeneration
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Limbal Stem-Cell Therapy and Long-Term Corneal Regeneration

Rama, Paolo ; Matuska, Stanislav ; Paganoni, Giorgio ; Spinelli, Alessandra ; De Luca, Michele ; Pellegrini, Graziella

The New England journal of medicine, 2010-07, Vol.363 (2), p.147-155 [Periódico revisado por pares]

Waltham, MA: Massachusetts Medical Society

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3
Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector : Short-Term Results of a Phase I Trial
Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector : Short-Term Results of a Phase I Trial
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Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector : Short-Term Results of a Phase I Trial

HAUSWIRTH, William W ; ALEMAN, Tomas S ; JACOBSON, Samuel G ; KAUSHAL, Shalesh ; CIDECIYAN, Artur V ; SCHWARTZ, Sharon B ; LILI WANG ; CONLON, Thomas J ; BOYE, Sanford L ; FLOTTE, Terence R ; BYRNE, Barry J

Human gene therapy, 2008-10, Vol.19 (10), p.979-990 [Periódico revisado por pares]

Larchmont, NY: Liebert

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4
Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease
Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease
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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

Human gene therapy, 2012-04, Vol.23 (4), p.377-381 [Periódico revisado por pares]

Larchmont, NY: Liebert

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5
Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates
Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates
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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

Human gene therapy, 2012-04, Vol.23 (4), p.382-389 [Periódico revisado por pares]

Larchmont, NY: Liebert

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6
Cardiopoietic Stem Cell Therapy in Heart Failure: The C-CURE (Cardiopoietic stem Cell therapy in heart failURE) Multicenter Randomized Trial With Lineage-Specified Biologics
Cardiopoietic Stem Cell Therapy in Heart Failure: The C-CURE (Cardiopoietic stem Cell therapy in heart failURE) Multicenter Randomized Trial With Lineage-Specified Biologics
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Cardiopoietic Stem Cell Therapy in Heart Failure: The C-CURE (Cardiopoietic stem Cell therapy in heart failURE) Multicenter Randomized Trial With Lineage-Specified Biologics

BARTUNEK, Jozef ; BEHFAR, Atta ; LEGRAND, Victor ; VRINTS, Christian ; VANOVERSCHELDE, Jean Louis ; CRESPO-DIAZ, Ruben ; HOMSY, Christian ; TENDERA, Michal ; WALDMAN, Scott ; WIJNS, William ; TERZIC, Andre ; DOLATABADI, Dariouch ; VANDERHEYDEN, Marc ; OSTOJIC, Miodrag ; DENS, Jo ; EL NAKADI, Badih ; BANOVIC, Marko ; BELESLIN, Branko ; VROLIX, Mathias

Journal of the American College of Cardiology, 2013-06, Vol.61 (23), p.2329-2338 [Periódico revisado por pares]

New York, NY: Elsevier

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7
Development of a novel systemic gene delivery system for cancer therapy with a tumor-specific cleavable PEG-lipid
Development of a novel systemic gene delivery system for cancer therapy with a tumor-specific cleavable PEG-lipid
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Development of a novel systemic gene delivery system for cancer therapy with a tumor-specific cleavable PEG-lipid

HATAKEYAMA, H ; AKITA, H ; KOGURE, K ; OISHI, M ; NAGASAKI, Y ; KIHIRA, Y ; UENO, M ; KOBAYASHI, H ; KIKUCHI, H ; HARASHIMA, H

Gene therapy, 2007-01, Vol.14 (1), p.68-77 [Periódico revisado por pares]

Basingstoke: Nature Publishing Group

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8
Gene therapy for red-green colour blindness in adult primates
Gene therapy for red-green colour blindness in adult primates
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Gene therapy for red-green colour blindness in adult primates

Mancuso, Katherine ; Neitz, Jay ; Neitz, Maureen ; Hauswirth, William W ; Connor, Thomas B ; Li, Qiuhong ; Kuchenbecker, James A ; Mauck, Matthew C

Nature (London), 2009-10, Vol.461 (7265), p.784-787 [Periódico revisado por pares]

London: Nature Publishing Group

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9
Targeted Delivery of Self-Complementary Adeno-Associated Virus Serotype 9 to the Brain, Using Magnetic Resonance Imaging-Guided Focused Ultrasound
Targeted Delivery of Self-Complementary Adeno-Associated Virus Serotype 9 to the Brain, Using Magnetic Resonance Imaging-Guided Focused Ultrasound
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Targeted Delivery of Self-Complementary Adeno-Associated Virus Serotype 9 to the Brain, Using Magnetic Resonance Imaging-Guided Focused Ultrasound

THEVENOT, Emmanuel ; JORDAO, Jessica F ; O'REILLY, Meaghan A ; MARKHAM, Kelly ; WENG, Ying-Qi ; FOUST, Kevin D ; KASPAR, Brian K ; HYNYNEN, Kullervo ; AUBERT, Isabelle

Human gene therapy, 2012-11, Vol.23 (11), p.1144-1155 [Periódico revisado por pares]

Larchmont, NY: Liebert

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10
Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia
Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia
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Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia

LI, C ; NARKBUNNAM, N ; SAMULSKI, R. J ; ASOKAN, A ; HU, G ; JACOBSON, L. J ; MANCO-JOHNSON, M. J ; MONAHAN, P. E

Gene therapy, 2012-03, Vol.19 (3), p.288-294 [Periódico revisado por pares]

London: Nature Publishing Group

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