| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models
|
Murakami, Yoshiko
|
|
|
32 |
1 |
p.
|
artikel
|
| 2 |
AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life
|
Doshi, Bhavya S.
|
|
|
32 |
1 |
p.
|
artikel
|
| 3 |
AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich’s ataxia
|
Chang, Joshua C.
|
|
|
32 |
1 |
p.
|
artikel
|
| 4 |
A gene silencing-based approach to tackle fatty liver disease
|
Strnad, Pavel
|
|
|
32 |
1 |
p.
|
artikel
|
| 5 |
All-in-one IQ toggle switches with high versatilities for fine-tuning of transgene expression in mammalian cells and tissues
|
Hong, Jeongkwan
|
|
|
32 |
1 |
p.
|
artikel
|
| 6 |
A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR
|
Zhang, Junping
|
|
|
32 |
1 |
p.
|
artikel
|
| 7 |
A novel FOXP3 knockout-humanized mouse model for pre-clinical safety and efficacy evaluation of Treg-like cell products
|
Sato, Yohei
|
|
|
32 |
1 |
p.
|
artikel
|
| 8 |
A positive take on negative selection for CAR-T manufacturing
|
Orentas, Rimas J.
|
|
|
32 |
1 |
p.
|
artikel
|
| 9 |
B cell focused transient immune suppression protocol for efficient AAV readministration to the liver
|
Rana, Jyoti
|
|
|
32 |
1 |
p.
|
artikel
|
| 10 |
Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques
|
Blackwood, Meghan
|
|
|
32 |
1 |
p.
|
artikel
|
| 11 |
Can mitochondria brown the lower-limb adipocytes?
|
Doulamis, Ilias P.
|
|
|
32 |
1 |
p.
|
artikel
|
| 12 |
Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice
|
Smith, Miles C.
|
|
|
32 |
1 |
p.
|
artikel
|
| 13 |
Continuous manufacturing of lentiviral vectors using a stable producer cell line in a fixed-bed bioreactor
|
Stibbs, Dale J.
|
|
|
32 |
1 |
p.
|
artikel
|
| 14 |
Efficient and sustained FOXP3 locus editing in hematopoietic stem cells as a therapeutic approach for IPEX syndrome
|
Singh, Swati
|
|
|
32 |
1 |
p.
|
artikel
|
| 15 |
Expanded specific T cells to hypomutated regions of the SARS-CoV-2 using mRNA electroporated antigen-presenting cells
|
Ogando-Rivas, Elizabeth
|
|
|
32 |
1 |
p.
|
artikel
|
| 16 |
Extracellular vesicle depletion and UGCG overexpression mitigate the cell density effect in HEK293 cell culture transfection
|
Pérez-Rubio, Pol
|
|
|
32 |
1 |
p.
|
artikel
|
| 17 |
Extracellular vesicles ameliorates sleep deprivation induced anxiety-like behavior and cognitive impairment in mice
|
Lin, Yiqin
|
|
|
32 |
1 |
p.
|
artikel
|
| 18 |
First use of adeno-associated viruses in the human inner ear
|
Landegger, Lukas D.
|
|
|
32 |
1 |
p.
|
artikel
|
| 19 |
Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome
|
Pille, Melissa
|
|
|
32 |
1 |
p.
|
artikel
|
| 20 |
Genetic surgery for a cystic fibrosis-causing splicing mutation
|
Bulcaen, Mattijs
|
|
|
32 |
1 |
p.
|
artikel
|
| 21 |
Genome editing in clinical practice: A model study for next-gen hematopoietic cell transplants in hematologic malignancies
|
Derigs, Patrick
|
|
|
32 |
1 |
p.
|
artikel
|
| 22 |
Gesicles packaging dCas9-VPR ribonucleoprotein complexes can combine with vorinostat and promote HIV proviral transcription
|
Fisher, Michaela A.
|
|
|
32 |
1 |
p.
|
artikel
|
| 23 |
Glutaredoxin-1 modulates the NF-κB signaling pathway to activate inducible nitric oxide synthase in experimental necrotizing enterocolitis
|
Zhang, Yunfei
|
|
|
32 |
1 |
p.
|
artikel
|
| 24 |
High-titer manufacturing of SARS-CoV-2 Spike-pseudotyped VSV in stirred-tank bioreactors
|
Todesco, Hayley M.
|
|
|
32 |
1 |
p.
|
artikel
|
| 25 |
Improving cell-specific recombination using AAV vectors in the murine CNS by capsid and expression cassette optimization
|
Kawabata, Hayato
|
|
|
32 |
1 |
p.
|
artikel
|
| 26 |
Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study
|
Ryckman, Alex E.
|
|
|
32 |
1 |
p.
|
artikel
|
| 27 |
Large-scale generation of IL-12 secreting macrophages from human pluripotent stem cells for cancer therapy
|
Kang, Baoqiang
|
|
|
32 |
1 |
p.
|
artikel
|
| 28 |
Lipid nanoparticle-encapsulated DNA vaccine robustly induce superior immune responses to the mRNA vaccine in Syrian hamsters
|
Liao, Hung-Chun
|
|
|
32 |
1 |
p.
|
artikel
|
| 29 |
Liter-scale manufacturing of shelf-stable plasmid DNA/PEI transfection particles for viral vector production
|
Hu, Yizong
|
|
|
32 |
1 |
p.
|
artikel
|
| 30 |
Long-term stability of clinical-grade lentiviral vectors for cell therapy
|
Jadlowsky, Julie K.
|
|
|
32 |
1 |
p.
|
artikel
|
| 31 |
Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency
|
Mével, Mathieu
|
|
|
32 |
1 |
p.
|
artikel
|
| 32 |
Manufacture of CD22 CAR T cells following positive versus negative selection results in distinct cytokine secretion profiles and γδ T cell output
|
Song, Hannah W.
|
|
|
32 |
1 |
p.
|
artikel
|
| 33 |
Measurement solutions and standards for advanced therapy
|
Wang, Lili
|
|
|
32 |
1 |
p.
|
artikel
|
| 34 |
Navigating toward gene therapy in Marfan syndrome: A hope for halting aortic aneurysm
|
Egea, Gustavo
|
|
|
32 |
1 |
p.
|
artikel
|
| 35 |
New perspectives for gene therapy of the X-linked form of Charcot-Marie-Tooth disease
|
Caballé, Rafael Balada
|
|
|
32 |
1 |
p.
|
artikel
|
| 36 |
Non-canonical capsid engineering highlights new possibilities for AAV vectorology
|
Zhang, Zehan
|
|
|
32 |
1 |
p.
|
artikel
|
| 37 |
Nonviral delivery of nCas9 for “safe harbor” integration to treat MPS IVA
|
Ou, Li
|
|
|
32 |
1 |
p.
|
artikel
|
| 38 |
Optimization of anti-CD19 CAR T cell production for treatment of patients with chronic lymphocytic leukemia
|
Amatya, Christina
|
|
|
32 |
1 |
p.
|
artikel
|
| 39 |
Optimizing lentiviral genomic integrations to cure beta-thalassemia: The least required for success?
|
Rivella, Stefano
|
|
|
32 |
1 |
p.
|
artikel
|
| 40 |
Perspectives of the Friedreich ataxia community on gene therapy clinical trials
|
Trantham, Shandra J.
|
|
|
32 |
1 |
p.
|
artikel
|
| 41 |
Preexisting antibody assays for gene therapy: Considerations on patient selection cutoffs and companion diagnostic requirements
|
Braun, Manuela
|
|
|
32 |
1 |
p.
|
artikel
|
| 42 |
Pulmonary macrophage transplant therapy in parenchymal lung diseases
|
O’Callaghan, Marissa
|
|
|
32 |
1 |
p.
|
artikel
|
| 43 |
Rationally engineered novel AAV capsids for intra-articular gene delivery
|
Li, Wenjun
|
|
|
32 |
1 |
p.
|
artikel
|
| 44 |
Recombinant AAV genome size effect on viral vector production, purification, and thermostability
|
Ibreljic, Nermin
|
|
|
32 |
1 |
p.
|
artikel
|
| 45 |
Regnase-1 overexpression as a therapeutic approach of Marfan syndrome
|
Noormalal, Marie
|
|
|
32 |
1 |
p.
|
artikel
|
| 46 |
TAZing down metabolic mayhem: siRNAs against liver inflammation and fibrosis in humanized mice
|
Weihs, Julian
|
|
|
32 |
1 |
p.
|
artikel
|
| 47 |
The comparability tales: A phase-appropriate roadmap for CGT drug product development
|
Clément, Nathalie
|
|
|
32 |
1 |
p.
|
artikel
|
| 48 |
The very stable lentiviral vector
|
Binder, Gwendolyn K.
|
|
|
32 |
1 |
p.
|
artikel
|
| 49 |
Thorough molecular configuration analysis of noncanonical AAV genomes in AAV vector preparations
|
Zhang, Junping
|
|
|
32 |
1 |
p.
|
artikel
|
| 50 |
Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74
|
Potter, Rachael A.
|
|
|
32 |
1 |
p.
|
artikel
|
| 51 |
Whole-body galactose oxidation as a robust functional assay to assess the efficacy of gene-based therapies in a mouse model of Galactosemia
|
Balakrishnan, Bijina
|
|
|
32 |
1 |
p.
|
artikel
|
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