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A largely random AAV integration profile after LPLD gene therapy
by Kaeppel, Christine
Nature medicine, 2013-07, Vol.19 (7), p.889-891
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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
by HAUSWIRTH, William W
Human gene therapy, 2008, Vol.19 (10), p.979-990
3.
Efficient Clinical Scale Gene Modification via Zinc Finger Nuclease–Targeted Disruption of the HIV Co-receptor CCR5
by Maier, Dawn A
Human gene therapy, 2013-03-01, Vol.24 (3), p.245-258
4.
A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear
by Landegger, Lukas D
Nature biotechnology, 2017-03, Vol.35 (3), p.280-284
5.
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies
by Leborgne, Christian
Nature Medicine, 2020-07, Vol.26 (7), p.1096-1101
6.
In utero CRISPR-mediated therapeutic editing of metabolic genes
by Rossidis, Avery C
Nature medicine, 2018-10, Vol.24 (10), p.1513-1518
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Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo
by Zufferey, Romain
Nature biotechnology, 1997-09, Vol.15 (9), p.871-875
8.
Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial
by Palfi, Stéphane, Prof
The Lancet (British edition), 2014, Vol.383 (9923), p.1138-1146
9.
Small But Increasingly Mighty: Latest Advances in AAV Vector Research, Design, and Evolution
by Grimm, Dirk
Human gene therapy, 2017-11-01, Vol.28 (11), p.175-1086
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Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
by Deverman, Benjamin E
Nature biotechnology, 2016-02, Vol.34 (2), p.204-209
11.
Comparison of Adeno-Associated Viral Vector Serotypes for Spinal Cord and Motor Neuron Gene Delivery
by Snyder, Brooke R
Human gene therapy, 2011-09-01, Vol.22 (9), p.1129-1135
12.
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
by Maguire, Albert M, MD
The Lancet (British edition), 2009, Vol.374 (9701), p.1597-1605
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Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success—A Personal Perspective
by Hastie, Eric
Human gene therapy, 2015-05-01, Vol.26 (5), p.257-265
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Newcastle disease virus (NDV) recombinant expressing the hemagglutinin of H7N9 avian influenza virus protects chickens against NDV and highly pathogenic avian influenza A (H7N9) vi...
by Hu, Zenglei
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Adenovirus: The First Effective In Vivo Gene Delivery Vector
by Crystal, Ronald G
Human gene therapy, 2014-01-01, Vol.25 (1), p.3-11
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A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome
by Winner, Leanne K
Human gene therapy, 2016-05-01, Vol.27 (5), p.363-375
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Immune Responses to Intramuscular Administration of Alipogene Tiparvovec (AAV1-LPLS447X) in a Phase II Clinical Trial of Lipoprotein Lipase Deficiency Gene Therapy
by Ferreira, Valerie
Human gene therapy, 2014-03-01, Vol.25 (3), p.18-188
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Plasmid DNA Manufacturing for Indirect and Direct Clinical Applications
by Schmeer, Marco
Human gene therapy, 2017-10-01, Vol.28 (10), p.856-861
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Targeted Delivery of Self-Complementary Adeno-Associated Virus Serotype 9 to the Brain, Using Magnetic Resonance Imaging-Guided Focused Ultrasound
by Thévenot, Emmanuel
Human gene therapy, 2012-11-01, Vol.23 (11), p.1144-1155
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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
by High, Katherine A
Nature medicine, 2006-03, Vol.12 (3), p.342-347
