| no |
title |
author |
magazine |
year |
volume |
issue |
page(s) |
type |
| 1 |
AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice
|
Doyle, Brendan M.
|
|
|
15 |
C |
p. 194-203
|
article
|
| 2 |
AAV-Mediated Expression of AP-1-Neutralizing RNA Decoy Oligonucleotides Attenuates Transplant Vasculopathy in Mouse Aortic Allografts
|
Remes, Anca
|
|
|
15 |
C |
p. 246-256
|
article
|
| 3 |
AAV-Mediated Gene Augmentation Therapy Restores Critical Functions in Mutant PRPF31+/− iPSC-Derived RPE Cells
|
Brydon, Elizabeth M.
|
|
|
15 |
C |
p. 392-402
|
article
|
| 4 |
AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes
|
Keskin, Sonay
|
|
|
15 |
C |
p. 275-284
|
article
|
| 5 |
ALPPL2 Is a Potential Diagnostic Biomarker for Pancreatic Cancer-Derived Extracellular Vesicles
|
Shin, Hye-Su
|
|
|
15 |
C |
p. 204-210
|
article
|
| 6 |
A Muscle Hybrid Promoter as a Novel Tool for Gene Therapy
|
Piekarowicz, Katarzyna
|
|
|
15 |
C |
p. 157-169
|
article
|
| 7 |
A Sendai Virus-Based Cytoplasmic RNA Vector as a Novel Platform for Long-Term Expression of MicroRNAs
|
Sano, Masayuki
|
|
|
15 |
C |
p. 371-382
|
article
|
| 8 |
Bi-allelic Homology-Directed Repair with Helper-Dependent Adenoviruses
|
Palmer, Donna J.
|
|
|
15 |
C |
p. 285-293
|
article
|
| 9 |
Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID
|
Charrier, Sabine
|
|
|
15 |
C |
p. 232-245
|
article
|
| 10 |
Clinical Considerations for Capsid Choice in the Development of Liver-Targeted AAV-Based Gene Transfer
|
Pipe, Steven
|
|
|
15 |
C |
p. 170-178
|
article
|
| 11 |
Developing an Anion Exchange Chromatography Assay for Determining Empty and Full Capsid Contents in AAV6.2
|
Wang, Chunlei
|
|
|
15 |
C |
p. 257-263
|
article
|
| 12 |
Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease
|
Martier, Raygene
|
|
|
15 |
C |
p. 343-358
|
article
|
| 13 |
Disease Modification by Combinatorial Single Vector Gene Therapy: A Preclinical Translational Study in Epilepsy
|
Melin, Esbjörn
|
|
|
15 |
C |
p. 179-193
|
article
|
| 14 |
Enhanced Factor IX Activity following Administration of AAV5-R338L “Padua” Factor IX versus AAV5 WT Human Factor IX in NHPs
|
Spronck, Elisabeth A.
|
|
|
15 |
C |
p. 221-231
|
article
|
| 15 |
Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA
|
Sorrentino, Nicolina Cristina
|
|
|
15 |
C |
p. 333-342
|
article
|
| 16 |
Gene Therapy Correction of Aldehyde Dehydrogenase 2 Deficiency
|
Matsumura, Yuki
|
|
|
15 |
C |
p. 72-82
|
article
|
| 17 |
HIV-Specific T Cell Responses Are Highly Stable on Antiretroviral Therapy
|
Xu, Yinyan
|
|
|
15 |
C |
p. 9-17
|
article
|
| 18 |
Immunology of Adenoviral Vectors in Cancer Therapy
|
Shaw, Amanda Rosewell
|
|
|
15 |
C |
p. 418-429
|
article
|
| 19 |
Improved GaLV-TR Glycoproteins to Pseudotype Lentiviral Vectors: Impact of Viral Protease Activity in the Production of LV Pseudotypes
|
Tomás, Hélio A.
|
|
|
15 |
C |
p. 1-8
|
article
|
| 20 |
Intrinsic Myogenic Potential of Skeletal Muscle-Derived Pericytes from Patients with Myotonic Dystrophy Type 1
|
Ausems, Cornelia Rosanne Maria
|
|
|
15 |
C |
p. 120-132
|
article
|
| 21 |
Lentiviral Vector Purification Using Nanofiber Ion-Exchange Chromatography
|
Ruscic, Jelena
|
|
|
15 |
C |
p. 52-62
|
article
|
| 22 |
Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1
|
Prabhakar, Shilpa
|
|
|
15 |
C |
p. 18-26
|
article
|
| 23 |
Low-Dose Busulfan Reduces Human CD34+ Cell Doses Required for Engraftment in c-kit Mutant Immunodeficient Mice
|
Leonard, Alexis
|
|
|
15 |
C |
p. 430-437
|
article
|
| 24 |
Machine Learning Enables Accurate Prediction of Asparagine Deamidation Probability and Rate
|
Delmar, Jared A.
|
|
|
15 |
C |
p. 264-274
|
article
|
| 25 |
mRNA as a Novel Treatment Strategy for Hereditary Spastic Paraplegia Type 5
|
Hauser, Stefan
|
|
|
15 |
C |
p. 359-370
|
article
|
| 26 |
Nanobody-Enhanced Targeting of AAV Gene Therapy Vectors
|
Eichhoff, Anna Marei
|
|
|
15 |
C |
p. 211-220
|
article
|
| 27 |
Non-antibiotic Small-Molecule Regulation of DHFR-Based Destabilizing Domains In Vivo
|
Peng, Hui
|
|
|
15 |
C |
p. 27-39
|
article
|
| 28 |
Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease
|
Kang, Hye-Ri
|
|
|
15 |
C |
p. 383-391
|
article
|
| 29 |
Phase 1 Safety and Immunogenicity Trial of Recombinant Lactococcus lactis Expressing Human Papillomavirus Type 16 E6 Oncoprotein Vaccine
|
Taghinezhad-S, Sedigheh
|
|
|
15 |
C |
p. 40-51
|
article
|
| 30 |
Plasmid-Mediated Gene Therapy in Mouse Models of Limb Girdle Muscular Dystrophy
|
Guha, Tuhin K.
|
|
|
15 |
C |
p. 294-304
|
article
|
| 31 |
Preclinical Evaluation of a Cell-Based Gene Therapy Using the Sleeping Beauty Transposon System in Choroidal Neovascularization
|
Hernandez, Maria
|
|
|
15 |
C |
p. 403-417
|
article
|
| 32 |
Preclinical Proof-of-Concept, Analytical Development, and Commercial Scale Production of Lentiviral Vector in Adherent Cells
|
Leinonen, Hanna M.
|
|
|
15 |
C |
p. 63-71
|
article
|
| 33 |
rAAVrh74.MCK.GALGT2 Demonstrates Safety and Widespread Muscle Glycosylation after Intravenous Delivery in C57BL/6J Mice
|
Zygmunt, Deborah A.
|
|
|
15 |
C |
p. 305-319
|
article
|
| 34 |
Removal of Endotoxin from rAAV Samples Using a Simple Detergent-Based Protocol
|
Kondratova, Liudmyla
|
|
|
15 |
C |
p. 112-119
|
article
|
| 35 |
Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle
|
Poupiot, Jérôme
|
|
|
15 |
C |
p. 83-100
|
article
|
| 36 |
Safety of Same-Eye Subretinal Sequential Readministration of AAV2-hRPE65v2 in Non-human Primates
|
Weed, Lindsey
|
|
|
15 |
C |
p. 133-148
|
article
|
| 37 |
Selection of an Efficient AAV Vector for Robust CNS Transgene Expression
|
Hanlon, Killian S.
|
|
|
15 |
C |
p. 320-332
|
article
|
| 38 |
Targeting Adeno-Associated Virus Vectors for Local Delivery to Fractures and Systemic Delivery to the Skeleton
|
Lee, Lucinda R.
|
|
|
15 |
C |
p. 101-111
|
article
|
| 39 |
Universal Template-Assisted, Cloning-free Method for the Generation of Small RNA-Expressing Dumbbell-Shaped DNA Vectors
|
Cyrill, Samantha Leeanne
|
|
|
15 |
C |
p. 149-156
|
article
|
Journal description