nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('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 |
artikel |
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 |
artikel |
3 |
AAV-Mediated Gene Augmentation Therapy Restores Critical Functions in Mutant PRPF31+/− iPSC-Derived RPE Cells
|
Brydon, Elizabeth M. |
|
|
15 |
C |
p. 392-402 |
artikel |
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 |
artikel |
5 |
ALPPL2 Is a Potential Diagnostic Biomarker for Pancreatic Cancer-Derived Extracellular Vesicles
|
Shin, Hye-Su |
|
|
15 |
C |
p. 204-210 |
artikel |
6 |
A Muscle Hybrid Promoter as a Novel Tool for Gene Therapy
|
Piekarowicz, Katarzyna |
|
|
15 |
C |
p. 157-169 |
artikel |
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 |
artikel |
8 |
Bi-allelic Homology-Directed Repair with Helper-Dependent Adenoviruses
|
Palmer, Donna J. |
|
|
15 |
C |
p. 285-293 |
artikel |
9 |
Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID
|
Charrier, Sabine |
|
|
15 |
C |
p. 232-245 |
artikel |
10 |
Clinical Considerations for Capsid Choice in the Development of Liver-Targeted AAV-Based Gene Transfer
|
Pipe, Steven |
|
|
15 |
C |
p. 170-178 |
artikel |
11 |
Developing an Anion Exchange Chromatography Assay for Determining Empty and Full Capsid Contents in AAV6.2
|
Wang, Chunlei |
|
|
15 |
C |
p. 257-263 |
artikel |
12 |
Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease
|
Martier, Raygene |
|
|
15 |
C |
p. 343-358 |
artikel |
13 |
Disease Modification by Combinatorial Single Vector Gene Therapy: A Preclinical Translational Study in Epilepsy
|
Melin, Esbjörn |
|
|
15 |
C |
p. 179-193 |
artikel |
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 |
artikel |
15 |
Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA
|
Sorrentino, Nicolina Cristina |
|
|
15 |
C |
p. 333-342 |
artikel |
16 |
Gene Therapy Correction of Aldehyde Dehydrogenase 2 Deficiency
|
Matsumura, Yuki |
|
|
15 |
C |
p. 72-82 |
artikel |
17 |
HIV-Specific T Cell Responses Are Highly Stable on Antiretroviral Therapy
|
Xu, Yinyan |
|
|
15 |
C |
p. 9-17 |
artikel |
18 |
Immunology of Adenoviral Vectors in Cancer Therapy
|
Shaw, Amanda Rosewell |
|
|
15 |
C |
p. 418-429 |
artikel |
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 |
artikel |
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 |
artikel |
21 |
Lentiviral Vector Purification Using Nanofiber Ion-Exchange Chromatography
|
Ruscic, Jelena |
|
|
15 |
C |
p. 52-62 |
artikel |
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 |
artikel |
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 |
artikel |
24 |
Machine Learning Enables Accurate Prediction of Asparagine Deamidation Probability and Rate
|
Delmar, Jared A. |
|
|
15 |
C |
p. 264-274 |
artikel |
25 |
mRNA as a Novel Treatment Strategy for Hereditary Spastic Paraplegia Type 5
|
Hauser, Stefan |
|
|
15 |
C |
p. 359-370 |
artikel |
26 |
Nanobody-Enhanced Targeting of AAV Gene Therapy Vectors
|
Eichhoff, Anna Marei |
|
|
15 |
C |
p. 211-220 |
artikel |
27 |
Non-antibiotic Small-Molecule Regulation of DHFR-Based Destabilizing Domains In Vivo
|
Peng, Hui |
|
|
15 |
C |
p. 27-39 |
artikel |
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 |
artikel |
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 |
artikel |
30 |
Plasmid-Mediated Gene Therapy in Mouse Models of Limb Girdle Muscular Dystrophy
|
Guha, Tuhin K. |
|
|
15 |
C |
p. 294-304 |
artikel |
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 |
artikel |
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 |
artikel |
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 |
artikel |
34 |
Removal of Endotoxin from rAAV Samples Using a Simple Detergent-Based Protocol
|
Kondratova, Liudmyla |
|
|
15 |
C |
p. 112-119 |
artikel |
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 |
artikel |
36 |
Safety of Same-Eye Subretinal Sequential Readministration of AAV2-hRPE65v2 in Non-human Primates
|
Weed, Lindsey |
|
|
15 |
C |
p. 133-148 |
artikel |
37 |
Selection of an Efficient AAV Vector for Robust CNS Transgene Expression
|
Hanlon, Killian S. |
|
|
15 |
C |
p. 320-332 |
artikel |
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 |
artikel |
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 |
artikel |