| no |
title |
author |
magazine |
year |
volume |
issue |
page(s) |
type |
| 1 |
AAV vectors engineered to target insulin receptor greatly enhance intramuscular gene delivery
|
Jackson, Cody B.
|
|
|
19 |
C |
p. 496-506
|
article
|
| 2 |
Adipose Tissue: An Emerging Target for Adeno-associated Viral Vectors
|
Bates, Rhiannon
|
|
|
19 |
C |
p. 236-249
|
article
|
| 3 |
A Generic Assay to Detect Aberrant ARSB Splicing and mRNA Degradation for the Molecular Diagnosis of MPS VI
|
Broeders, Mike
|
|
|
19 |
C |
p. 174-185
|
article
|
| 4 |
A Methodology for Assessing Tumor Clonality of Adult T Cell Leukemia/Lymphoma
|
Yamakawa, Tomohiro
|
|
|
19 |
C |
p. 467-473
|
article
|
| 5 |
A Novel Proteomic Method Reveals NLS Tagging of T-DM1 Contravenes Classical Nuclear Transport in a Model of HER2-Positive Breast Cancer
|
Lacasse, Vincent
|
|
|
19 |
C |
p. 99-119
|
article
|
| 6 |
A qPCR Method for AAV Genome Titer with ddPCR-Level of Accuracy and Precision
|
Wang, Yu
|
|
|
19 |
C |
p. 341-346
|
article
|
| 7 |
Characterization of AAV-Specific Affinity Ligands: Consequences for Vector Purification and Development Strategies
|
Mietzsch, Mario
|
|
|
19 |
C |
p. 362-373
|
article
|
| 8 |
Chemically Defined, High-Density Insect Cell-Based Expression System for Scalable AAV Vector Production
|
Kurasawa, James H.
|
|
|
19 |
C |
p. 330-340
|
article
|
| 9 |
Clinical Development of Gene Therapies: The First Three Decades and Counting
|
Lapteva, Larissa
|
|
|
19 |
C |
p. 387-397
|
article
|
| 10 |
CNS Transduction Benefits of AAV-PHP.eB over AAV9 Are Dependent on Administration Route and Mouse Strain
|
Mathiesen, Sophie N.
|
|
|
19 |
C |
p. 447-458
|
article
|
| 11 |
Conditionally Replicating Vectors Mobilize Chimeric Antigen Receptors against HIV
|
Urak, Ryan Z.
|
|
|
19 |
C |
p. 285-294
|
article
|
| 12 |
CRISPR-Mediated Base Conversion Allows Discriminatory Depletion of Endogenous T Cell Receptors for Enhanced Synthetic Immunity
|
Preece, Roland
|
|
|
19 |
C |
p. 149-161
|
article
|
| 13 |
Development and Optimization of a Hydrophobic Interaction Chromatography-Based Method of AAV Harvest, Capture, and Recovery
|
McNally, David J.
|
|
|
19 |
C |
p. 275-284
|
article
|
| 14 |
Development of a Novel Competitive qRT-PCR Assay to Measure Relative Lentiviral Packaging Efficiency
|
Vamva, Eirini
|
|
|
19 |
C |
p. 307-319
|
article
|
| 15 |
Effective In Vivo Gene Modification in Mouse Tissue-Resident Peritoneal Macrophages by Intraperitoneal Delivery of Lentiviral Vectors
|
Ipseiz, Natacha
|
|
|
19 |
C |
p. 375
|
article
|
| 16 |
Engineered Antigen-Specific T Cells Secreting Broadly Neutralizing Antibodies: Combining Innate and Adaptive Immune Response against HIV
|
Powell, Allison B.
|
|
|
19 |
C |
p. 78-88
|
article
|
| 17 |
Engineering and In Vitro Selection of a Novel AAV3B Variant with High Hepatocyte Tropism and Reduced Seroreactivity
|
Biswas, Moanaro
|
|
|
19 |
C |
p. 347-361
|
article
|
| 18 |
Envelope-Specific Adaptive Immunity following Transplantation of Hematopoietic Stem Cells Modified with VSV-G Lentivirus
|
Rust, Blake J.
|
|
|
19 |
C |
p. 438-446
|
article
|
| 19 |
Ex Vivo Gene Therapy Treats Bone Complications of Mucopolysaccharidosis Type II Mouse Models through Bone Remodeling Reactivation
|
Wada, Miho
|
|
|
19 |
C |
p. 261-274
|
article
|
| 20 |
High Levels of Frataxin Overexpression Lead to Mitochondrial and Cardiac Toxicity in Mouse Models
|
Belbellaa, Brahim
|
|
|
19 |
C |
p. 120-138
|
article
|
| 21 |
Identification of hsa-miR-1275 as a Novel Biomarker Targeting MECP2 for Human Epilepsy of Unknown Etiology
|
Zhao, Ye
|
|
|
19 |
C |
p. 398-410
|
article
|
| 22 |
In Situ Detection of Adeno-associated Viral Vector Genomes with SABER-FISH
|
Wang, Sean K.
|
|
|
19 |
C |
p. 376-386
|
article
|
| 23 |
Investigational Assay for Haplotype Phasing of the Huntingtin Gene
|
Svrzikapa, Nenad
|
|
|
19 |
C |
p. 162-173
|
article
|
| 24 |
In Vitro and In Vivo Amenability to Migalastat in Fabry Disease
|
Lenders, Malte
|
|
|
19 |
C |
p. 24-34
|
article
|
| 25 |
In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9
|
López-Manzaneda, Sergio
|
|
|
19 |
C |
p. 426-437
|
article
|
| 26 |
In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy
|
Kwon, Jennifer B.
|
|
|
19 |
C |
p. 320-329
|
article
|
| 27 |
Laboratory-Scale Lentiviral Vector Production and Purification for Enhanced Ex Vivo and In Vivo Genetic Engineering
|
Soldi, Monica
|
|
|
19 |
C |
p. 411-425
|
article
|
| 28 |
Lentiviral Vector Production from a Stable Packaging Cell Line Using a Packed Bed Bioreactor
|
Powers, Alicia D.
|
|
|
19 |
C |
p. 1-13
|
article
|
| 29 |
Long-Term Metabolic Correction of Phenylketonuria by AAV-Delivered Phenylalanine Amino Lyase
|
Tao, Rui
|
|
|
19 |
C |
p. 507-517
|
article
|
| 30 |
Machine Learning Enables Accurate Prediction of Asparagine Deamidation Probability and Rate
|
Delmar, Jared A.
|
|
|
19 |
C |
p. 374
|
article
|
| 31 |
Minimal Essential Human Factor VIII Alterations Enhance Secretion and Gene Therapy Efficiency
|
Cao, Wenjing
|
|
|
19 |
C |
p. 486-495
|
article
|
| 32 |
Modeling Transient Abnormal Myelopoiesis Using Induced Pluripotent Stem Cells and CRISPR/Cas9 Technology
|
Barwe, Sonali P.
|
|
|
19 |
C |
p. 201-209
|
article
|
| 33 |
MTA1, a Target of Resveratrol, Promotes Epithelial-Mesenchymal Transition of Endometriosis via ZEB2
|
Kong, Xiangyi
|
|
|
19 |
C |
p. 295-306
|
article
|
| 34 |
Nonclinical Studies that Support Viral Vector-Delivered Gene Therapies: An EFPIA Gene Therapy Working Group Perspective
|
Bolt, Michael W.
|
|
|
19 |
C |
p. 89-98
|
article
|
| 35 |
Optimization of S. aureus dCas9 and CRISPRi Elements for a Single Adeno-Associated Virus that Targets an Endogenous Gene
|
Backstrom, Jon R.
|
|
|
19 |
C |
p. 139-148
|
article
|
| 36 |
Rapid Lentiviral Vector Producer Cell Line Generation Using a Single DNA Construct
|
Chen, Yu Hua
|
|
|
19 |
C |
p. 47-57
|
article
|
| 37 |
Regeneration of Tumor-Antigen-Specific Cytotoxic T Lymphocytes from iPSCs Transduced with Exogenous TCR Genes
|
Maeda, Takuya
|
|
|
19 |
C |
p. 250-260
|
article
|
| 38 |
Repeat Dosing of AAV2.5T to Ferret Lungs Elicits an Antibody Response That Diminishes Transduction in an Age-Dependent Manner
|
Tang, Yinghua
|
|
|
19 |
C |
p. 186-200
|
article
|
| 39 |
Safety and Efficacy of an Immune Cell-Specific Chimeric Promoter in Regulating Anti-PD-1 Antibody Expression in CAR T Cells
|
Fang, Yuan
|
|
|
19 |
C |
p. 14-23
|
article
|
| 40 |
Synthetic Prostacyclin Agonist Attenuates Pressure-Overloaded Cardiac Fibrosis by Inhibiting FMT
|
Masada, Kenta
|
|
|
19 |
C |
p. 210-219
|
article
|
| 41 |
Targeting the Root Cause of Mucopolysaccharidosis IIIA with a New scAAV9 Gene Replacement Vector
|
Bobo, Tierra A.
|
|
|
19 |
C |
p. 474-485
|
article
|
| 42 |
Tetravalent Bispecific Tandem Antibodies Improve Brain Exposure and Efficacy in an Amyloid Transgenic Mouse Model
|
Do, Tuan-Minh
|
|
|
19 |
C |
p. 58-77
|
article
|
| 43 |
The Effect of Residual Triton X-100 on Structural Stability and Infection Activity of Adenovirus Particles
|
Ma, Jinhu
|
|
|
19 |
C |
p. 35-46
|
article
|
| 44 |
Transduction of Salivary Gland Acinar Cells with a Novel AAV Vector 44.9
|
Di Pasquale, Giovanni
|
|
|
19 |
C |
p. 459-466
|
article
|
| 45 |
WAS Promoter-Driven Lentiviral Vectors Mimic Closely the Lopsided WASP Expression during Megakaryocytic Differentiation
|
Muñoz, Pilar
|
|
|
19 |
C |
p. 220-235
|
article
|
Journal description