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#	Title	Journal	Year	Citations
1	Recombinant AAV Viral Vectors Pseudotyped with Viral Capsids from Serotypes 1, 2, and 5 Display Differential Efficiency and Cell Tropism after Delivery to Different Regions of the Central Nervous System	Molecular Therapy	2004	669
2	Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors	Methods	2002	514
3	AAV-Mediated Gene Therapy for Research and Therapeutic Purposes	Annual Review of Virology	2014	400
4	Current Clinical Applications of In Vivo Gene Therapy with AAVs	Molecular Therapy	2021	380
5	Gene Therapy with Brain-Derived Neurotrophic Factor As a Protection: Retinal Ganglion Cells in a Rat Glaucoma Model		2003	336
6	HumanRPE65Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year	Human Gene Therapy	2009	305
7	Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy	Proceedings of the National Academy of Sciences of the United States of America	2009	295
8	Identification of Amino Acid Residues in the Capsid Proteins of Adeno-Associated Virus Type 2 That Contribute to Heparan Sulfate Proteoglycan Binding	Journal of Virology	2003	294
9	Small Molecule Regulators of Protein Arginine Methyltransferases	Journal of Biological Chemistry	2004	281
10	Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors	Gene Therapy	2001	272
11	A Comprehensive Review of Retinal Gene Therapy	Molecular Therapy	2013	245
12	Novel Properties of Tyrosine-mutant AAV2 Vectors in the Mouse Retina	Molecular Therapy	2011	234
13	Sustained peripheral expression of transgene adiponectin offsets the development of diet-induced obesity in rats	Proceedings of the National Academy of Sciences of the United States of America	2003	227
14	Manufacturing of recombinant adeno-associated viral vectors for clinical trials	Molecular Therapy - Methods and Clinical Development	2016	198
15	Safety of Recombinant Adeno-Associated Virus Type 2–RPE65 Vector Delivered by Ocular Subretinal Injection	Molecular Therapy	2006	196
16	Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial	Human Genetics	2016	195
17	Kinetic Analyses of Stability of Simple and Complex Retroviral Vectors	Virology	2001	169
18	Colocalization of muscleblind with RNA foci is separable from mis-regulation of alternative splicing in myotonic dystrophy	Journal of Cell Science	2005	168
19	Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Serotype 2α1-Antitrypsin (AAT) Vector in AAT-Deficient Adults	Human Gene Therapy	2006	168
20	Adeno-Associated Virus Type 2 VP2 Capsid Protein Is Nonessential and Can Tolerate Large Peptide Insertions at Its N Terminus	Journal of Virology	2004	167
21	CMV-β-Actin Promoter Directs Higher Expression from an Adeno-Associated Viral Vector in the Liver than the Cytomegalovirus or Elongation Factor 1α Promoter and Results in Therapeutic Levels of Human Factor X in Mice	Human Gene Therapy	2001	163
22	Safety in Nonhuman Primates of Ocular AAV2-RPE65, a Candidate Treatment for Blindness in Leber Congenital Amaurosis	Human Gene Therapy	2006	142
23	IL-10 Suppresses Chemokines, Inflammation, and Fibrosis in a Model of Chronic Renal Disease	Journal of the American Society of Nephrology: JASN	2005	134
24	Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort	Annals of Neurology	2016	131
25	Systemic Overexpression of IL-10 Induces CD4+CD25+ Cell Populations In Vivo and Ameliorates Type 1 Diabetes in Nonobese Diabetic Mice in a Dose-Dependent Fashion	Journal of Immunology	2003	125
26	Differential transgene expression in brain cells in vivo and in vitro from AAV-2 vectors with small transcriptional control units	Virology	2003	124
27	Successful Production of Pseudotyped rAAV Vectors Using a Modified Baculovirus Expression System	Molecular Therapy	2005	116
28	Developmental expression of mouse muscleblind genes Mbnl1, Mbnl2 and Mbnl3	Gene Expression Patterns	2003	113
29	Adeno-Associated Virus-Vectored Gene Therapy for Retinal Disease	Human Gene Therapy	2005	98
30	Large-Scale Adeno-Associated Viral Vector Production Using a Herpesvirus-Based System Enables Manufacturing for Clinical Studies	Human Gene Therapy	2009	98
31	Recombinant adeno-associated virus-mediated alpha-1 antitrypsin gene therapy prevents type I diabetes in NOD mice	Gene Therapy	2004	97
32	Recombinant Adeno-Associated Viral Vectors in the Nervous System	Human Gene Therapy	2005	97
33	Glycosylation-independent Lysosomal Targeting of Acid α-Glucosidase Enhances Muscle Glycogen Clearance in Pompe Mice	Journal of Biological Chemistry	2013	97
34	Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial	The Lancet Child and Adolescent Health	2017	96
35	Structure of Adeno-Associated Virus Type 4	Journal of Virology	2005	95
36	Magnetic Resonance Imaging and Spectroscopy Assessment of Lower Extremity Skeletal Muscles in Boys with Duchenne Muscular Dystrophy: A Multicenter Cross Sectional Study	PLoS ONE	2014	94
37	DNA-dependent PK inhibits adeno-associated virus DNA integration	Proceedings of the National Academy of Sciences of the United States of America	2004	91
38	Nigrostriatal rAAV-mediated GDNF Overexpression Induces Robust Weight Loss in a Rat Model of Age-related Obesity	Molecular Therapy	2009	91
39	Optic Neuropathy Induced by Reductions in Mitochondrial Superoxide Dismutase		2003	90
40	Intramuscular Administration of Recombinant Adeno-Associated Virus 2 α-1 Antitrypsin (rAAV-SERPINA1) Vectors in a Nonhuman Primate Model: Safety and Immunologic Aspects	Molecular Therapy	2002	87
41	Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: Effects of serotype and site of administration	Vision Research	2008	87
42	Lentiviral siRNAs targeting multiple highly conserved RNA sequences of human immunodeficiency virus type 1	Gene Therapy	2005	85
43	Preclinical Potency and Safety Studies of an AAV2-Mediated Gene Therapy Vector for the Treatment of MERTK Associated Retinitis Pigmentosa	Human Gene Therapy Clinical Development	2013	84
44	Physiological Correction of Pompe Disease by Systemic Delivery of Adeno-associated Virus Serotype 1 Vectors	Molecular Therapy	2007	83
45	Efficient Ex Vivo Transduction of Pancreatic Islet Cells With Recombinant Adeno-Associated Virus Vectors	Diabetes	2001	81
46	α1-Antitrypsin Gene Therapy Modulates Cellular Immunity and Efficiently Prevents Type 1 Diabetes in Nonobese Diabetic Mice	Human Gene Therapy	2006	81
47	Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice	Genetic Vaccines and Therapy	2008	80
48	A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency	Brain	2018	80
49	Tadalafil Alleviates Muscle Ischemia in Patients with Becker Muscular Dystrophy	Science Translational Medicine	2012	79
50	Systemic Mannitol-Induced Hyperosmolality Amplifies rAAV2-Mediated Striatal Transduction to a Greater Extent Than Local Co-infusion	Molecular Therapy	2005	74

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