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50 results found

no	title	author	magazine	year	volume	issue	page(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	article
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	article
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	article
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	article
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	article
6	A Digestion-free Method for Quantification of Residual Host Cell DNA in rAAV Gene Therapy Products	Wang, Yu			13	C	p. 526-531	article
7	Advanced Technology for Gene Delivery with Homing Peptides to Spinal Cord through Systemic Circulation in Mice	Terashima, Tomoya			13	C	p. 474-483	article
8	Advancements and Obstacles of CRISPR-Cas9 Technology in Translational Research	You, Liting			13	C	p. 359-370	article
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	article
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	article
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	article
12	Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia	Kang, Hye-Ri			13	C	p. 265-273	article
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	article
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	article
15	CRISPR Activation Enhances In Vitro Potency of AAV Vectors Driven by Tissue-Specific Promoters	McDougald, Devin S.			13	C	p. 380-389	article
16	Endothelin-1 Augments Therapeutic Potency of Human Mesenchymal Stem Cells via CDH2 and VEGF Signaling	Lee, Eun Ju			13	C	p. 503-511	article
17	Engineering Synthetic Chromosomes by Sequential Loading of Multiple Genomic Payloads over 100 Kilobase Pairs in Size	Greene, Amy			13	C	p. 463-473	article
18	EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation	Detela, Giulia			13	C	p. 205-232	article
19	Generate TALE/TALEN as Easily and Rapidly as Generating CRISPR	Zhang, Shuyan			13	C	p. 310-320	article
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	article
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	article
22	Highly Efficient and Selective CAR-Gene Transfer Using CD4- and CD8-Targeted Lentiviral Vectors	Jamali, Arezoo			13	C	p. 371-379	article
23	Hippocampal GAD67 Transduction Using rAAV8 Regulates Epileptogenesis in EL Mice	Shimazaki, Kuniko			13	C	p. 180-186	article
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	article
25	Improved Loading of Plasma-Derived Extracellular Vesicles to Encapsulate Antitumor miRNAs	Pomatto, Margherita Alba Carlotta			13	C	p. 133-144	article
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	article
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	article
28	Investigational RNAi Therapeutic Targeting C5 Is Efficacious in Pre-clinical Models of Myasthenia Gravis	Kusner, Linda L.			13	C	p. 484-492	article
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	article
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	article
31	Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates	Ohno, Kousaku			13	C	p. 47-54	article
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	article
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	article
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	article
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	article
36	Novel Diagnostic Tool for p47 phox -Deficient Chronic Granulomatous Disease Patient and Carrier Detection	Wrona, Dominik			13	C	p. 274-278	article
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	article
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	article
39	Preclinical Testing in Translational Animal Models of Prader-Willi Syndrome: Overview and Gap Analysis	Carias, K. Vanessa			13	C	p. 344-358	article
40	Production of CRISPR/Cas9-Mediated Self-Cleaving Helper-Dependent Adenoviruses	Palmer, Donna J.			13	C	p. 432-439	article
41	Quantifying Antigen-Specific T Cell Responses When Using Antigen-Agnostic Immunotherapies	van Vloten, Jacob P.			13	C	p. 154-166	article
42	Rapid AAV-Neutralizing Antibody Determination with a Cell-Binding Assay	Guo, Ping			13	C	p. 40-46	article
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	article
44	Scalable Production of AAV Vectors in Orbitally Shaken HEK293 Cells	Blessing, Daniel			13	C	p. 14-26	article
45	Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I	Ghosh, Arunabha			13	C	p. 321-333	article
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	article
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	article
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	article
49	Trans-ocular Electric Current In Vivo Enhances AAV-Mediated Retinal Gene Transduction after Intravitreal Vector Administration	Song, Hongman			13	C	p. 77-85	article
50	Utilization of HEPES for Enhancing Protein Transfection into Mammalian Cells	Chen, Shun-Hua			13	C	p. 99-111	article

50 results found

Koninklijke Bibliotheek - National Library of the Netherlands