D. Mccarty

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Limited Entry of Adenovirus Vectors into Well-Differentiated Airway Epithelium Is Responsible for Inefficient Gene Transfer

S. Randell, R. Boucher, R. Pickles, 1998, Journal of Virology.

Self-complementary AAV vectors; advances and applications.

D. Mccarty, 2008, Molecular therapy : the journal of the American Society of Gene Therapy.

Integration of adeno-associated virus (AAV) and recombinant AAV vectors.

R. Samulski, D. Mccarty, S. M. Young, 2004, Annual review of genetics.

Host Cell DNA Repair Pathways in Adeno-Associated Viral Genome Processing

V. Choi, R. Samulski, D. Mccarty, 2006, Journal of Virology.

Adeno‐associated virus serotypes 1 to 5 mediated tumor cell directed gene transfer and improvement of transduction efficiency

M. Hallek, R. Samulski, L. Perabò, 2005, The journal of gene medicine.

Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery.

H. Fu, D. Mccarty, S. Killedar, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

Sequences required for coordinate induction of adeno-associated virus p19 and p40 promoters by Rep protein

N. Muzyczka, D. Mccarty, M. Christensen, 1991, Journal of virology.

Differential distribution of heparan sulfate glycoforms and elevated expression of heparan sulfate biosynthetic enzyme genes in the brain of mucopolysaccharidosis IIIB mice

T. V. van Kuppevelt, H. Fu, D. Mccarty, 2011, Metabolic Brain Disease.

Delivery of MDR1 small interfering RNA by self-complementary recombinant adeno-associated virus vector.

R. Samulski, Dong Xu, R. Juliano, 2005, Molecular therapy : the journal of the American Society of Gene Therapy.

Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice.

F. J. Duncan, K. Clark, H. Fu, 2015, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene Therapy for Neurodevelopmental Disorders

H. Fu, D. Mccarty, 2014 .

Peripheral Nervous System Neuropathology and Progressive Sensory Impairments in a Mouse Model of Mucopolysaccharidosis IIIB

H. Fu, D. Mccarty, R. Stephens, 2012, PloS one.

Chronic ethanol increases adeno‐associated viral transgene expression in rat liver via oxidant and NFκB‐dependent mechanisms

I. Rusyn, R. Samulski, G. Arteel, 2000, Hepatology.

Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice

J. Muenzer, S. Moy, A. Perez, 2007, Gene Therapy.

Oligodendrocyte‐specific gene expression in mouse brain: Use of a myelin‐forming cell type–specific promoter in an adeno‐associated virus

Hong Yu Chen, Kinuko Suzuki, D. Mccarty, 1999, Journal of neuroscience research.

New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors

Xiao Xiao, D. Mccarty, F. K. Ferrari, 1997, Nature Medicine.

Restoration of central nervous system α‐N‐acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno‐associated viral type 2 vector delivery

J. Muenzer, H. Fu, D. Mccarty, 2010, The journal of gene medicine.

The adeno-associated virus (AAV) Rep protein acts as both a repressor and an activator to regulate AAV transcription during a productive infection

N. Muzyczka, D. Mccarty, D. J. Pereira, 1997, Journal of virology.

Targeting Root Cause by Systemic scAAV9-hIDS Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice

J. Muenzer, H. Fu, D. Mccarty, 2018, Molecular therapy. Methods & clinical development.

Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates.

K. Flanigan, K. McBride, C. Walker, 2014, Human gene therapy. Clinical development.

In vitro replication of adeno-associated virus DNA

N. Muzyczka, I. Zolotukhin, X. Zhou, 1994, Journal of virology.

Factors Influencing Adeno-Associated Virus-Mediated Gene Transfer to Human Cystic Fibrosis Airway Epithelial Cells: Comparison with Adenovirus Vectors

R. Samulski, S. Teramoto, J. Bartlett, 1998, Journal of Virology.

A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB.

K. Flanigan, K. McBride, C. Walker, 2015, Human gene therapy. Clinical development.

Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating MPS IIIB: Toxicology, Bio-distribution and Immunological Assessments in Primates.

K. Flanigan, K. McBride, C. Walker, 2014, Human gene therapy. Clinical development.

Innate and adaptive immune activation in the brain of MPS IIIB mouse model

H. Auer, H. Fu, D. Mccarty, 2009, Journal of neuroscience research.

A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design.

K. Flanigan, K. McNally, K. McBride, 2016, Molecular genetics and metabolism.

Crossing the blood-brain-barrier with viral vectors.

H. Fu, D. Mccarty, 2016, Current opinion in virology.

Immunogenicity and inflammatory properties of respiratory syncytial virus attachment G protein in cotton rats

M. Peeples, D. Mccarty, S. Niewiesk, 2021, PloS one.

High-resolution gel and hybridization analysis of plant mitochondrial RNA

W. Hauswirth, D. Mccarty, Brian S. Masters, 1983, Plant Molecular Biology Reporter.

Sequence and transcriptional analysis of a chloroplast insert in the mitochondrial genome ofZea mays

W. Hauswirth, D. Mccarty, Brian S. Masters, 1988, Current Genetics.

Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

R. Dolmetsch, K. Eggan, R. Crystal, 2022, Molecular therapy : the journal of the American Society of Gene Therapy.

Effective Depletion of Pre-existing Anti-AAV Antibodies Requires Broad Immune Targeting

H. Fu, D. Mccarty, Aaron S. Meadows, 2016, Molecular therapy. Methods & clinical development.

Journal of Neuroinflammation Open Access Research Mucopolysaccharidosis Iiib, a Lysosomal Storage Disease, Triggers a Pathogenic Cns Autoimmune Response

P. Popovich, H. Fu, D. Mccarty, 2022 .

Researchdosis IIIB, a lysosomal storage disease, triggers a pathogenic CNS autoimmune response

P. Popovich, H. Fu, D. Mccarty, 2010 .

Responsible for Inefficient Gene Transfer Well-Differentiated Airway Epithelium Is Limited Entry of Adenovirus Vectors into

S. Randell, R. Boucher, R. Pickles, 2013 .

Rapid transgene expression in multiple precursor cell types of adult rat subventricular zone mediated by adeno-associated type 1 vectors.

M. Levivier, O. De Witte, R. Samulski, 2012, Human gene therapy.

Identification of linear DNA sequences that specifically bind the adeno-associated virus Rep protein

J. Ryan, N. Muzyczka, Xiaohuai Zhou, 1994, Journal of virology.

Interaction of the adeno-associated virus Rep protein with a sequence within the A palindrome of the viral terminal repeat

J. Ryan, N. Muzyczka, S. Zolotukhin, 1994, Journal of virology.

Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats

J. Morrison, W. Janssen, M. Banck, 2006, Molecular pain.

Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity.

K. L. La Perle, H. Fu, D. Mccarty, 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

Analysis of mutations in adeno-associated virus Rep protein in vivo and in vitro

N. Muzyczka, D. Mccarty, T. Ni, 1992, Journal of virology.

Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery

M. Cataldi, H. Fu, D. Mccarty, 2016, Molecular therapy. Methods & clinical development.

Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery.

R. Mohney, H. Fu, D. Mccarty, 2017, Molecular Therapy.

Lentivirus vector purification using anion exchange HPLC leads to improved gene transfer.

V. Madden, D. Mccarty, C. Walsh, 2003, BioTechniques.

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

R. Samulski, P. Monahan, H. Fu, 2003, Gene Therapy.

Roles of Adeno-Associated Virus Rep Protein and Human Chromosome 19 in Site-Specific Recombination

R. Samulski, N. Degtyareva, D. Mccarty, 2000, Journal of Virology.

Biodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles

I. Pardo, X. Palazzi, L. Whiteley, 2021, Human gene therapy.

Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy.

K. Flanigan, K. McBride, H. Fu, 2017, Human gene therapy. Clinical development.

Differential prevalence of antibodies against adeno-associated virus in healthy children and patients with mucopolysaccharidosis III: perspective for AAV-mediated gene therapy.

K. Flanigan, K. McBride, H. Fu, 2017, Human gene therapy. Clinical development.

Pre-Clinical Safety Study Feasibility and Safety of Systemic rAAV 9h NAGLU Delivery for Treating Mucopolysaccharidosis IIIB : Toxicology , Biodistribution , and Immunological Assessments in Primates

K. Flanigan, K. McBride, C. Walker, 2014 .

Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain.

R. Samulski, J. Muenzer, H. Fu, 2003, Molecular therapy : the journal of the American Society of Gene Therapy.

Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice

J. Muenzer, H. Fu, D. Mccarty, 2009, Gene Therapy.

Blood genome-wide transcriptional profiles reflect broad molecular impairments and strong blood-brain links in Alzheimer's disease.

D. Scharre, N. Stoicea, H. Fu, 2014, Journal of Alzheimer's disease : JAD.

Serum global metabolomics profiling reveals profound metabolic impairments in patients with MPS IIIA and MPS IIIB

S. Stirdivant, R. Mohney, H. Fu, 2017, Metabolic Brain Disease.

Amyloidosis, Synucleinopathy, and Prion Encephalopathy in a Neuropathic Lysosomal Storage Disease: The CNS-Biomarker Potential of Peripheral Blood

H. Fu, D. Mccarty, Aaron S. Meadows, 2013, PloS one.

OPTICS 2901 Quantum cascade laser: Temperature dependence of the performance characteristics and high T0 operation

Stephen R. Forrest, Koji Tominaga, Carlo Sirtori, 1994 .

Durability of transgene expression after rAAV gene therapy

G. Gao, D. Levy, D. Mccarty, 2022, Molecular therapy : the journal of the American Society of Gene Therapy.

Effects of Adeno-Associated Virus DNA Hairpin Structure on Recombination

V. Choi, R. Samulski, D. Mccarty, 2005, Journal of Virology.

Development and Characterization of Novel Empty Adenovirus Capsids and Their Impact on Cellular Gene Expression

Atsuko Negishi, Richard Superfine, R. Superfine, 2003, Journal of Virology.

Adeno-associated virus vectors for gene transfer into erythroid cells.

R. Samulski, D. Mccarty, 1996, Current topics in microbiology and immunology.

Hairpin end conformation of adeno-associated virus (AAV) genome determines interactions with DNA repair pathways

M. Cataldi, D. Mccarty, 2012, Gene Therapy.

AAV hybrid serotypes: improved vectors for gene delivery.

V. Choi, R. Samulski, D. Mccarty, 2005, Current gene therapy.

Production of rAAV by plasmid transfection induces antiviral and inflammatory responses in suspension HEK293 cells

C. Chung, V. Wingate, D. Mccarty, 2023, Molecular therapy. Methods & clinical development.

Analysis of the interaction between adeno-associated virus and heparan sulfate using atomic force microscopy.

R. Superfine, Atsuko Negishi, R. Samulski, 2004, Glycobiology.

Identification and Characterization of an Adeno-Associated Virus Integration Site in CV-1 Cells from the African Green Monkey

R. Samulski, D. Mccarty, T. J. Amiss, 2003, Journal of Virology.

Replication-deficient adenoviral vector for gene transfer potentiates airway neurogenic inflammation.

R. Boucher, R. Pickles, D. Mccarty, 1997, American journal of respiratory cell and molecular biology.

CV-1 Cells from the African Green Monkey Adeno-Associated Virus Integration Site in Identification and Characterization of an

R. Samulski, D. Mccarty, T. J. Amiss, 2002 .

Nucleotide sequence of the Zea mays mitochondrial cytochrome oxidase subunit III gene.

W. Hauswirth, D. Mccarty, G. Hehman, 1988, Nucleic acids research.

Mucopolysaccharidosis IIIB, a lysosomal storage disease, triggers a pathogenic CNS autoimmune response

P. Popovich, H. Fu, D. Mccarty, 2010 .

Differential Effects of DNA Double-Strand Break Repair Pathways on Single-Strand and Self-Complementary Adeno-Associated Virus Vector Genomes

M. Cataldi, D. Mccarty, 2010, Journal of Virology.

Crossing the blood-brain-barrier with viral vectors.

H. Fu, D. Mccarty, 2016, Current opinion in virology.

Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery.

R. Mohney, H. Fu, D. Mccarty, 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

Innate and adaptive immune activation in the brain of MPS IIIB mouse model

H. Auer, H. Fu, D. Mccarty, 2009, Journal of Neuroscience Research.