| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
A consolidated AAV system for single-cut CRISPR correction of a common Duchenne muscular dystrophy mutation
|
Zhang, Yu
|
|
|
22 |
C |
p. 122-132
|
artikel
|
| 2 |
Antigen modifications improve nucleoside-modified mRNA-based influenza virus vaccines in mice
|
Freyn, Alec W.
|
|
|
22 |
C |
p. 84-95
|
artikel
|
| 3 |
A sensitive and reproducible cell-based assay via secNanoLuc to detect neutralizing antibody against adeno-associated virus vector capsid
|
Baatartsogt, Nemekhbayar
|
|
|
22 |
C |
p. 162-171
|
artikel
|
| 4 |
AUF1 gene transfer increases exercise performance and improves skeletal muscle deficit in adult mice
|
Abbadi, Dounia
|
|
|
22 |
C |
p. 222-236
|
artikel
|
| 5 |
Cardiotoxicity assessment using 3D vascularized cardiac tissue consisting of human iPSC-derived cardiomyocytes and fibroblasts
|
Tadano, Kiyoshi
|
|
|
22 |
C |
p. 338-349
|
artikel
|
| 6 |
Characterization and modulation of anti-αβTCR antibodies and their respective binding sites at the βTCR chain to enrich engineered T cells
|
Kierkels, Guido J.J.
|
|
|
22 |
C |
p. 388-400
|
artikel
|
| 7 |
Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina
|
Chandler, Laurel C.
|
|
|
22 |
C |
p. 52-65
|
artikel
|
| 8 |
Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency
|
Fañanas-Baquero, Sara
|
|
|
22 |
C |
p. 237-248
|
artikel
|
| 9 |
Complete intra-laboratory validation of a LAL assay for bacterial endotoxin determination in EBV-specific cytotoxic T lymphocytes
|
Pasqua, Salvatore
|
|
|
22 |
C |
p. 320-329
|
artikel
|
| 10 |
Depletion of high-content CD14+ cells from apheresis products is critical for successful transduction and expansion of CAR T cells during large-scale cGMP manufacturing
|
Wang, Xiuyan
|
|
|
22 |
C |
p. 377-387
|
artikel
|
| 11 |
Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia
|
Wang, Lili
|
|
|
22 |
C |
p. 1-10
|
artikel
|
| 12 |
Directed evolution of AAV accounting for long-term and enhanced transduction of cardiovascular endothelial cells in vivo
|
Liu, Y.B.
|
|
|
22 |
C |
p. 148-161
|
artikel
|
| 13 |
Efficacious, safe, and stable inhibition of corneal neovascularization by AAV-vectored anti-VEGF therapeutics
|
Su, Wenqi
|
|
|
22 |
C |
p. 107-121
|
artikel
|
| 14 |
Evidence generation and reproducibility in cell and gene therapy research: A call to action
|
Abou-el-Enein, Mohamed
|
|
|
22 |
C |
p. 11-14
|
artikel
|
| 15 |
Exosomes and organ-specific metastasis
|
Zhao, Lei
|
|
|
22 |
C |
p. 133-147
|
artikel
|
| 16 |
Formatting and gene-based delivery of a human PD-L1 single domain antibody for immune checkpoint blockade
|
Awad, Robin Maximilian
|
|
|
22 |
C |
p. 172-182
|
artikel
|
| 17 |
Gene supplementation of CYP27A1 in the liver restores bile acid metabolism in a mouse model of cerebrotendinous xanthomatosis
|
Lumbreras, Sara
|
|
|
22 |
C |
p. 210-221
|
artikel
|
| 18 |
Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral abnormalities and EEG activity
|
Hooper, Alexander W.M.
|
|
|
22 |
C |
p. 196-209
|
artikel
|
| 19 |
ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia
|
Ilyinskii, Petr O.
|
|
|
22 |
C |
p. 279-292
|
artikel
|
| 20 |
Immune inactivation of anti-simian immunodeficiency virus chimeric antigen receptor T cells in rhesus macaques
|
Haeseleer, Françoise
|
|
|
22 |
C |
p. 304-319
|
artikel
|
| 21 |
Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes
|
Sevilla, Julián
|
|
|
22 |
C |
p. 66-75
|
artikel
|
| 22 |
LATE–a novel sensitive cell-based assay for the study of CRISPR/Cas9-related long-term adverse treatment effects
|
Głów, Dawid
|
|
|
22 |
C |
p. 249-262
|
artikel
|
| 23 |
Low endotoxin E. coli strain-derived plasmids reduce rAAV vector-mediated immune responses both in vitro and in vivo
|
Zheng, Qingyun
|
|
|
22 |
C |
p. 293-303
|
artikel
|
| 24 |
Monitoring cell-mediated immune responses in AAV gene therapy clinical trials using a validated IFN-γ ELISpot method
|
Patton, Kathryn S.
|
|
|
22 |
C |
p. 183-195
|
artikel
|
| 25 |
Monolayer platform using human biopsy-derived duodenal organoids for pharmaceutical research
|
Yamashita, Tomoki
|
|
|
22 |
C |
p. 263-278
|
artikel
|
| 26 |
Outcomes of progranulin gene therapy in the retina are dependent on time and route of delivery
|
Zin, Emilia A.
|
|
|
22 |
C |
p. 40-51
|
artikel
|
| 27 |
Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series
|
Canarutto, Daniele
|
|
|
22 |
C |
p. 76-83
|
artikel
|
| 28 |
Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment
|
Navarro, Susana
|
|
|
22 |
C |
p. 350-359
|
artikel
|
| 29 |
Pre-retinal delivery of recombinant adeno-associated virus vector significantly improves retinal transduction efficiency
|
Zhang, Hanmeng
|
|
|
22 |
C |
p. 96-106
|
artikel
|
| 30 |
Reproducible immortalization of erythroblasts from multiple stem cell sources provides approach for sustainable RBC therapeutics
|
Daniels, Deborah E.
|
|
|
22 |
C |
p. 26-39
|
artikel
|
| 31 |
Substantial restoration of night vision in adult mice with congenital stationary night blindness
|
Varin, Juliette
|
|
|
22 |
C |
p. 15-25
|
artikel
|
| 32 |
Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1
|
Sahenk, Zarife
|
|
|
22 |
C |
p. 401-414
|
artikel
|
| 33 |
Treatment with bone maturation and average lifespan of HPP model mice by AAV8-mediated neonatal gene therapy via single muscle injection
|
Matsumoto, Tae
|
|
|
22 |
C |
p. 330-337
|
artikel
|
| 34 |
Unique somatic variants in DNA from urine exosomes of individuals with bladder cancer
|
Zhou, Xunian
|
|
|
22 |
C |
p. 360-376
|
artikel
|
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