| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAV2/8 Anti-angiogenic Gene Therapy Using Single-Chain Antibodies Inhibits Murine Choroidal Neovascularization
|
Hughes, Chris P.
|
|
|
13 |
C |
p. 86-98
|
artikel
|
| 2 |
AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models
|
Spronck, Elisabeth A.
|
|
|
13 |
C |
p. 334-343
|
artikel
|
| 3 |
AAV9-TAZ Gene Replacement Ameliorates Cardiac TMT Proteomic Profiles in a Mouse Model of Barth Syndrome
|
Suzuki-Hatano, Silveli
|
|
|
13 |
C |
p. 167-179
|
artikel
|
| 4 |
Achieving High-Yield Production of Functional AAV5 Gene Delivery Vectors via Fedbatch in an Insect Cell-One Baculovirus System
|
Joshi, Pranav R.H.
|
|
|
13 |
C |
p. 279-289
|
artikel
|
| 5 |
Adeno-Associated Viral Vector 2 and 9 Transduction Is Enhanced in Streptozotocin-Induced Diabetic Mouse Retina
|
Lee, Si Hyung
|
|
|
13 |
C |
p. 55-66
|
artikel
|
| 6 |
A Digestion-free Method for Quantification of Residual Host Cell DNA in rAAV Gene Therapy Products
|
Wang, Yu
|
|
|
13 |
C |
p. 526-531
|
artikel
|
| 7 |
Advanced Technology for Gene Delivery with Homing Peptides to Spinal Cord through Systemic Circulation in Mice
|
Terashima, Tomoya
|
|
|
13 |
C |
p. 474-483
|
artikel
|
| 8 |
Advancements and Obstacles of CRISPR-Cas9 Technology in Translational Research
|
You, Liting
|
|
|
13 |
C |
p. 359-370
|
artikel
|
| 9 |
An AAV-SGCG Dose-Response Study in a γ-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress
|
Israeli, David
|
|
|
13 |
C |
p. 494-502
|
artikel
|
| 10 |
Anti-cancer Effects of a Chemically Modified miR-143 on Bladder Cancer by Either Systemic or Intravesical Treatment
|
Yoshikawa, Yuki
|
|
|
13 |
C |
p. 290-302
|
artikel
|
| 11 |
Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease
|
Wang, Gensheng
|
|
|
13 |
C |
p. 493
|
artikel
|
| 12 |
Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia
|
Kang, Hye-Ri
|
|
|
13 |
C |
p. 265-273
|
artikel
|
| 13 |
Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study
|
Gruntman, Alisha M.
|
|
|
13 |
C |
p. 233-242
|
artikel
|
| 14 |
CD46 Null Packaging Cell Line Improves Measles Lentiviral Vector Production and Gene Delivery to Hematopoietic Stem and Progenitor Cells
|
Ozog, Stosh
|
|
|
13 |
C |
p. 27-39
|
artikel
|
| 15 |
CRISPR Activation Enhances In Vitro Potency of AAV Vectors Driven by Tissue-Specific Promoters
|
McDougald, Devin S.
|
|
|
13 |
C |
p. 380-389
|
artikel
|
| 16 |
Endothelin-1 Augments Therapeutic Potency of Human Mesenchymal Stem Cells via CDH2 and VEGF Signaling
|
Lee, Eun Ju
|
|
|
13 |
C |
p. 503-511
|
artikel
|
| 17 |
Engineering Synthetic Chromosomes by Sequential Loading of Multiple Genomic Payloads over 100 Kilobase Pairs in Size
|
Greene, Amy
|
|
|
13 |
C |
p. 463-473
|
artikel
|
| 18 |
EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation
|
Detela, Giulia
|
|
|
13 |
C |
p. 205-232
|
artikel
|
| 19 |
Generate TALE/TALEN as Easily and Rapidly as Generating CRISPR
|
Zhang, Shuyan
|
|
|
13 |
C |
p. 310-320
|
artikel
|
| 20 |
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
|
György, Bence
|
|
|
13 |
C |
p. 1-13
|
artikel
|
| 21 |
High-Efficiency Lentiviral Transduction of Human CD34+ Cells in High-Density Culture with Poloxamer and Prostaglandin E2
|
Uchida, Naoya
|
|
|
13 |
C |
p. 187-196
|
artikel
|
| 22 |
Highly Efficient and Selective CAR-Gene Transfer Using CD4- and CD8-Targeted Lentiviral Vectors
|
Jamali, Arezoo
|
|
|
13 |
C |
p. 371-379
|
artikel
|
| 23 |
Hippocampal GAD67 Transduction Using rAAV8 Regulates Epileptogenesis in EL Mice
|
Shimazaki, Kuniko
|
|
|
13 |
C |
p. 180-186
|
artikel
|
| 24 |
Human Galectin-1 Improves Sarcolemma Stability and Muscle Vascularization in the mdx Mouse Model of Duchenne Muscular Dystrophy
|
Wuebbles, Ryan D.
|
|
|
13 |
C |
p. 145-153
|
artikel
|
| 25 |
Improved Loading of Plasma-Derived Extracellular Vesicles to Encapsulate Antitumor miRNAs
|
Pomatto, Margherita Alba Carlotta
|
|
|
13 |
C |
p. 133-144
|
artikel
|
| 26 |
In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver
|
Liefhebber, Jolanda M.
|
|
|
13 |
C |
p. 512-525
|
artikel
|
| 27 |
Induction of Expandable Tissue-Specific Progenitor Cells from Human Pancreatic Tissue through Transient Expression of Defined Factors
|
Noguchi, Hirofumi
|
|
|
13 |
C |
p. 243-252
|
artikel
|
| 28 |
Investigational RNAi Therapeutic Targeting C5 Is Efficacious in Pre-clinical Models of Myasthenia Gravis
|
Kusner, Linda L.
|
|
|
13 |
C |
p. 484-492
|
artikel
|
| 29 |
In Vitro Expansion of Anti-viral T Cells from Cord Blood by Accelerated Co-cultured Dendritic Cells
|
Kuranda, Klaudia
|
|
|
13 |
C |
p. 112-120
|
artikel
|
| 30 |
In Vitro Macrophage Assay Predicts the In Vivo Anti-inflammatory Potential of Exosomes from Human Mesenchymal Stromal Cells
|
Pacienza, Natalia
|
|
|
13 |
C |
p. 67-76
|
artikel
|
| 31 |
Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates
|
Ohno, Kousaku
|
|
|
13 |
C |
p. 47-54
|
artikel
|
| 32 |
Large-Scale Generation and Characterization of Homogeneous Populations of Migratory Cortical Interneurons from Human Pluripotent Stem Cells
|
Ni, Peiyan
|
|
|
13 |
C |
p. 414-430
|
artikel
|
| 33 |
Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction
|
Han, Sang-oh
|
|
|
13 |
C |
p. 431
|
artikel
|
| 34 |
miR-195 Has a Potential to Treat Ischemic and Hemorrhagic Stroke through Neurovascular Protection and Neurogenesis
|
Cheng, Hsin-Yun
|
|
|
13 |
C |
p. 121-132
|
artikel
|
| 35 |
MTBVAC-Based TB-HIV Vaccine Is Safe, Elicits HIV-T Cell Responses, and Protects against Mycobacterium tuberculosis in Mice
|
Broset, Esther
|
|
|
13 |
C |
p. 253-264
|
artikel
|
| 36 |
Novel Diagnostic Tool for p47 phox -Deficient Chronic Granulomatous Disease Patient and Carrier Detection
|
Wrona, Dominik
|
|
|
13 |
C |
p. 274-278
|
artikel
|
| 37 |
PGE2 and Poloxamer Synperonic F108 Enhance Transduction of Human HSPCs with a β-Globin Lentiviral Vector
|
Masiuk, Katelyn E.
|
|
|
13 |
C |
p. 390-398
|
artikel
|
| 38 |
Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA
|
Ellison, Stuart M.
|
|
|
13 |
C |
p. 399-413
|
artikel
|
| 39 |
Preclinical Testing in Translational Animal Models of Prader-Willi Syndrome: Overview and Gap Analysis
|
Carias, K. Vanessa
|
|
|
13 |
C |
p. 344-358
|
artikel
|
| 40 |
Production of CRISPR/Cas9-Mediated Self-Cleaving Helper-Dependent Adenoviruses
|
Palmer, Donna J.
|
|
|
13 |
C |
p. 432-439
|
artikel
|
| 41 |
Quantifying Antigen-Specific T Cell Responses When Using Antigen-Agnostic Immunotherapies
|
van Vloten, Jacob P.
|
|
|
13 |
C |
p. 154-166
|
artikel
|
| 42 |
Rapid AAV-Neutralizing Antibody Determination with a Cell-Binding Assay
|
Guo, Ping
|
|
|
13 |
C |
p. 40-46
|
artikel
|
| 43 |
Safety of CD34+ Hematopoietic Stem Cells and CD4+ T Lymphocytes Transduced with LVsh5/C46 in HIV-1 Infected Patients with High-Risk Lymphoma
|
Delville, Marianne
|
|
|
13 |
C |
p. 303-309
|
artikel
|
| 44 |
Scalable Production of AAV Vectors in Orbitally Shaken HEK293 Cells
|
Blessing, Daniel
|
|
|
13 |
C |
p. 14-26
|
artikel
|
| 45 |
Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I
|
Ghosh, Arunabha
|
|
|
13 |
C |
p. 321-333
|
artikel
|
| 46 |
Targeted Gene Delivery into the Mammalian Inner Ear Using Synthetic Serotypes of Adeno-Associated Virus Vectors
|
Kim, Min-A
|
|
|
13 |
C |
p. 197-204
|
artikel
|
| 47 |
The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy
|
Long, Brian R.
|
|
|
13 |
C |
p. 440-452
|
artikel
|
| 48 |
Threshold for Pre-existing Antibody Levels Limiting Transduction Efficiency of Systemic rAAV9 Gene Delivery: Relevance for Translation
|
Meadows, Aaron S.
|
|
|
13 |
C |
p. 453-462
|
artikel
|
| 49 |
Trans-ocular Electric Current In Vivo Enhances AAV-Mediated Retinal Gene Transduction after Intravitreal Vector Administration
|
Song, Hongman
|
|
|
13 |
C |
p. 77-85
|
artikel
|
| 50 |
Utilization of HEPES for Enhancing Protein Transfection into Mammalian Cells
|
Chen, Shun-Hua
|
|
|
13 |
C |
p. 99-111
|
artikel
|
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