| Emerging Issues in AAV-Mediated In Vivo Gene Therapy |
115 |
| Engineering and Design of Chimeric Antigen Receptors |
48 |
| Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors |
45 |
| Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population |
37 |
| Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction |
31 |
| Engineering Nanoparticles for Targeted Delivery of Nucleic Acid Therapeutics in Tumor |
30 |
| Improved Loading of Plasma-Derived Extracellular Vesicles to Encapsulate Antitumor miRNAs |
30 |
| Gene Transfer with AAV9-PHP.B Rescues Hearing in a Mouse Model of Usher Syndrome 3A and Transduces Hair Cells in a Non-human Primate |
26 |
| Pharmacology of Recombinant Adeno-associated Virus Production |
23 |
| EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation |
23 |
| Biocompatible, Purified VEGF-A mRNA Improves Cardiac Function after Intracardiac Injection 1 Week Post-myocardial Infarction in Swine |
21 |
| A Rapamycin-Based GMP-Compatible Process for the Isolation and Expansion of Regulatory T Cells for Clinical Trials |
19 |
| A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing |
19 |
| Optimizing EphA2-CAR T Cells for the Adoptive Immunotherapy of Glioma |
18 |
| Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques |
16 |
| An Introduction to the Analysis of Single-Cell RNA-Sequencing Data |
16 |
| The Pharmacology of T Cell Therapies |
16 |
| Challenges in Advanced Therapy Medicinal Product Development: A Survey among Companies in Europe |
15 |
| Prevalence of Anti-Adeno-Associated Virus Immune Responses in International Cohorts of Healthy Donors |
15 |
| Expression of Human ACE2 in Lactobacillus and Beneficial Effects in Diabetic Retinopathy in Mice |
15 |
| Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with beta-Thalassemia Major |
15 |
| miR-195 Has a Potential to Treat Ischemic and Hemorrhagic Stroke through Neurovascular Protection and Neurogenesis |
15 |
| Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes |
15 |
| Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy |
15 |
| AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice |
14 |
| Enhanced Expression of Anti-CD19 Chimeric Antigen Receptor in piggyBac Transposon-Engineered T Cells |
14 |
| Advancements and Obstacles of CRISPR-Cas9 Technology in Translational Research |
14 |
| Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques |
13 |
| Development of a Novel Anti-CD19 Chimeric Antigen Receptor: A Paradigm for an Affordable CAR T Cell Production at Academic Institutions |
13 |
| Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65 |
12 |
| Immunology of Adenoviral Vectors in Cancer Therapy |
12 |
| A Novel Triple-Mutant AAV6 Capsid Induces Rapid and Potent Transgene Expression in the Muscle and Respiratory Tract of Mice |
12 |
| HDAd5/35(++) Adenovirus Vector Expressing Anti-CRISPR Peptides Decreases CRISPR/Cas9 Toxicity in Human Hematopoietic Stem Cells |
12 |
| Rapamycin Rescues Age-Related Changes in Muscle-Derived Stem/Progenitor Cells from Progeroid Mice |
12 |
| Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs |
11 |
| Enhanced Production of Exosome-Associated AAV by Overexpression of the Tetraspanin CD9 |
11 |
| Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid alpha-Glucosidase RescuesBoth Muscle and CNS Defects in Murine Pompe Disease |
11 |
| Platelet-Targeted Gene Therapy for Hemophilia |
11 |
| Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A |
10 |
| Surface-Engineered Lentiviral Vectors for Selective Gene Transfer into Subtypes of Lymphocytes |
10 |
| Inferring Novel Tumor Suppressor Genes with a Protein-Protein Interaction Network and Network Diffusion Algorithms |
10 |
| AAV-8 and AAV-9 Vectors Cooperate with Serum Proteins Differently Than MV-1 and AAV-6 |
10 |
| A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates |
10 |
| Seizure-Suppressant and Neuroprotective Effects of Encapsulated BDNF-Producing Cells in a Rat Model of Temporal Lobe Epilepsy |
10 |
| Novel Chimeric Gene Therapy Vectors Based on Adeno-Associated Virus and Four Different Mammalian Bocaviruses |
10 |
| Targeted Gene Delivery into the Mammalian Inner Ear Using Synthetic Serotypes of Adeno-Associated Virus Vectors |
10 |
| Highly Efficient and Selective CAR-Gene Transfer Using CD4-and CD8-Targeted Lentiviral Vectors |
10 |
| Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application |
10 |
| Helper-free Production of Laboratory Grade AAV and Purification by lodixanol Density Gradient Centrifugation |
9 |
| Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD |
9 |
