| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAV-CRB2 protects against vision loss in an inducible CRB1 retinitis pigmentosa mouse model
|
Buck, Thilo M.
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20 |
C |
p. 423-441
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artikel
|
| 2 |
AAV8 locoregional delivery induces long-term expression of an immunogenic transgene in macaques despite persisting local inflammation
|
Gernoux, Gwladys
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20 |
C |
p. 660-674
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artikel
|
| 3 |
AAV9-mediated gene delivery of MCT1 to oligodendrocytes does not provide a therapeutic benefit in a mouse model of ALS
|
Eykens, Caroline
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20 |
C |
p. 508-519
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artikel
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| 4 |
Ad- and AAV8-mediated ABCA1 gene therapy in a murine model with retinal ischemia/reperfusion injuries
|
Luo, Jing
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20 |
C |
p. 551-558
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artikel
|
| 5 |
A human surfactant B deficiency air-liquid interface cell culture model suitable for gene therapy applications
|
Munis, Altar M.
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20 |
C |
p. 237-246
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artikel
|
| 6 |
Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies
|
Gimpel, Andreas L.
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20 |
C |
p. 740-754
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artikel
|
| 7 |
Applying machine learning to predict viral assembly for adeno-associated virus capsid libraries
|
Marques, Andrew D.
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20 |
C |
p. 276-286
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artikel
|
| 8 |
A universal protocol for isolating retinal ON bipolar cells across species via fluorescence-activated cell sorting
|
Murenu, Elisa
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20 |
C |
p. 587-600
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artikel
|
| 9 |
Automated generation of gene-edited CAR T cells at clinical scale
|
Alzubi, Jamal
|
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20 |
C |
p. 379-388
|
artikel
|
| 10 |
Broadly active zinc finger protein-guided transcriptional activation of HIV-1
|
Scott, Tristan A.
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20 |
C |
p. 18-29
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artikel
|
| 11 |
Cell therapy with hiPSC-derived RPE cells and RPCs prevents visual function loss in a rat model of retinal degeneration
|
Salas, Anna
|
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20 |
C |
p. 688-702
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artikel
|
| 12 |
Comparative engraftment and clonality of macaque HSPCs expanded on human umbilical vein endothelial cells versus non-expanded cells
|
Srivastava, Sandeep K.
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20 |
C |
p. 703-715
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artikel
|
| 13 |
Comprehensive and systemic optimization for improving the yield of SARS-CoV-2 spike pseudotyped virus
|
Fu, Xinping
|
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20 |
C |
p. 350-356
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artikel
|
| 14 |
Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector
|
Dahl, Maria
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20 |
C |
p. 312-323
|
artikel
|
| 15 |
CReVIS-Seq: A highly accurate and multiplexable method for genome-wide mapping of lentiviral integration sites
|
Kim, Heon Seok
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20 |
C |
p. 792-800
|
artikel
|
| 16 |
CRISPR-Cas9 gene editing of hepatitis B virus in chronically infected humanized mice
|
Stone, Daniel
|
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20 |
C |
p. 258-275
|
artikel
|
| 17 |
CRISPR/Cas9-mediated generation and analysis of N terminus polymorphic models of β2AR in isogenic hPSC-derived cardiomyocytes
|
Kondrashov, Alexander
|
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20 |
C |
p. 39-53
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artikel
|
| 18 |
CRISPR-mediated rapid generation of neural cell-specific knockout mice facilitates research in neurophysiology and pathology
|
Xiao, Dan
|
|
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20 |
C |
p. 755-764
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artikel
|
| 19 |
Deferoxamine mesylate improves splicing and GAA activity of the common c.-32-13T>G allele in late-onset PD patient fibroblasts
|
Buratti, Emanuele
|
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20 |
C |
p. 227-236
|
artikel
|
| 20 |
Development of a new genetic reference material system based on Saccharomyces cerevisiae cells
|
He, Xin
|
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|
20 |
C |
p. 473-482
|
artikel
|
| 21 |
Directed Evolution of AAV Serotype 5 for Increased Hepatocyte Transduction and Retained Low Humoral Seroreactivity
|
Qian, Randolph
|
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20 |
C |
p. 122-132
|
artikel
|
| 22 |
Effective, safe, and sustained correction of murine XLA using a UCOE-BTK promoter-based lentiviral vector
|
Seymour, Brenda J.
|
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20 |
C |
p. 635-651
|
artikel
|
| 23 |
Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients
|
Shi, Xiaoxia
|
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20 |
C |
p. 287-297
|
artikel
|
| 24 |
Efficient treatment of a preclinical inflammatory bowel disease model with engineered bacteria
|
Ferenczi, Szilamer
|
|
|
20 |
C |
p. 218-226
|
artikel
|
| 25 |
Elimination of Mutant mtDNA by an Optimized mpTALEN Restores Differentiation Capacities of Heteroplasmic MELAS-iPSCs
|
Yahata, Naoki
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20 |
C |
p. 54-68
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artikel
|
| 26 |
Enhanced expression of immune checkpoint receptors during SARS-CoV-2 viral infection
|
Saheb Sharif-Askari, Narjes
|
|
|
20 |
C |
p. 109-121
|
artikel
|
| 27 |
Enriching leukapheresis improves T cell activation and transduction efficiency during CAR T processing
|
Noaks, Elsa
|
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|
20 |
C |
p. 675-687
|
artikel
|
| 28 |
Evaluation of the human type 3 adenoviral dodecahedron as a vector to target acute myeloid leukemia
|
Caulier, Benjamin
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20 |
C |
p. 181-190
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artikel
|
| 29 |
Galactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of concept
|
Cadaoas, Jaclyn
|
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20 |
C |
p. 191-203
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artikel
|
| 30 |
GAMER-Ad: a novel and rapid method for generating recombinant adenoviruses
|
Hamdan, Firas
|
|
|
20 |
C |
p. 625-634
|
artikel
|
| 31 |
Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID
|
Carbonaro-Sarracino, Denise A.
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|
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20 |
C |
p. 765-778
|
artikel
|
| 32 |
Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal
|
Moscatelli, Ilana
|
|
|
20 |
C |
p. 389-397
|
artikel
|
| 33 |
Gene therapy strategies for idiopathic pulmonary fibrosis: recent advances, current challenges, and future directions
|
Ruigrok, Mitchel J.R.
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|
|
20 |
C |
p. 483-496
|
artikel
|
| 34 |
Genetic engineering of human and mouse CD4+ and CD8+ Tregs using lentiviral vectors encoding chimeric antigen receptors
|
Vimond, Nadège
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|
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20 |
C |
p. 69-85
|
artikel
|
| 35 |
Glycoengineering of AAV-delivered monoclonal antibodies yields increased ADCC activity
|
Termini, James M.
|
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20 |
C |
p. 204-217
|
artikel
|
| 36 |
Human-specific GAPDH qRT-PCR is an accurate and sensitive method of xenograft metastasis quantification
|
Dahn, Margaret L.
|
|
|
20 |
C |
p. 398-408
|
artikel
|
| 37 |
iMATCH: an integrated modular assembly system for therapeutic combination high-capacity adenovirus gene therapy
|
Brücher, Dominik
|
|
|
20 |
C |
p. 572-586
|
artikel
|
| 38 |
Improved diabetic wound healing by LFcinB is associated with relevant changes in the skin immune response and microbiota
|
Mouritzen, Michelle V.
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|
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20 |
C |
p. 726-739
|
artikel
|
| 39 |
Improved transduction of canine X-linked muscular dystrophy with rAAV9-microdystrophin via multipotent MSC pretreatment
|
Hayashita-Kinoh, Hiromi
|
|
|
20 |
C |
p. 133-141
|
artikel
|
| 40 |
Inhibition of indoleamine 2,3-dioxygenase 1 synergizes with oxaliplatin for efficient colorectal cancer therapy
|
Miao, Xiaofei
|
|
|
20 |
C |
p. 442-450
|
artikel
|
| 41 |
Inhibition of KDM1A activity restores adult neurogenesis and improves hippocampal memory in a mouse model of Kabuki syndrome
|
Zhang, Li
|
|
|
20 |
C |
p. 779-791
|
artikel
|
| 42 |
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice
|
White, Katherine A.
|
|
|
20 |
C |
p. 497-507
|
artikel
|
| 43 |
In vivo PCSK9 gene editing using an all-in-one self-cleavage AAV-CRISPR system
|
Li, Qian
|
|
|
20 |
C |
p. 652-659
|
artikel
|
| 44 |
Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector
|
De Sabbata, Giulia
|
|
|
20 |
C |
p. 169-180
|
artikel
|
| 45 |
Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors
|
Ferla, Rita
|
|
|
20 |
C |
p. 247-257
|
artikel
|
| 46 |
miR-1254 induced by NESG1 inactivates HDGF/DDX5-stimulated nuclear translocation of β-catenin and suppresses NPC metastasis
|
Cheng, Chao
|
|
|
20 |
C |
p. 615-624
|
artikel
|
| 47 |
Modulation of the liver immune microenvironment by the adeno-associated virus serotype 8 gene therapy vector
|
Carestia, Agostina
|
|
|
20 |
C |
p. 95-108
|
artikel
|
| 48 |
Molecular subtyping and functional validation of TTK, TPX2, UBE2C, and LRP8 in sensitivity of TNBC to paclitaxel
|
Elango, Ramesh
|
|
|
20 |
C |
p. 601-614
|
artikel
|
| 49 |
Monitoring of tisagenlecleucel transgene DNA using a quantitative polymerase chain reaction assay
|
Davis, Lisa
|
|
|
20 |
C |
p. 535-541
|
artikel
|
| 50 |
Non-viral ex vivo genome-editing in mouse bona fide hematopoietic stem cells with CRISPR/Cas9
|
Byambaa, Suvd
|
|
|
20 |
C |
p. 451-462
|
artikel
|
| 51 |
Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease
|
Francis, Jeremy S.
|
|
|
20 |
C |
p. 520-534
|
artikel
|
| 52 |
qPCR and qRT-PCR analysis: Regulatory points to consider when conducting biodistribution and vector shedding studies
|
Ma, Haiyan
|
|
|
20 |
C |
p. 152-168
|
artikel
|
| 53 |
Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning
|
Nonnenmacher, Mathieu
|
|
|
20 |
C |
p. 366-378
|
artikel
|
| 54 |
Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation
|
Counsell, John R.
|
|
|
20 |
C |
p. 357-365
|
artikel
|
| 55 |
Serum exosome microRNA panel as a noninvasive biomarker for molecular diagnosis of fulminant myocarditis
|
Zhang, Yingying
|
|
|
20 |
C |
p. 142-151
|
artikel
|
| 56 |
siRNA targeting Schlemm’s canal endothelial tight junctions enhances outflow facility and reduces IOP in a steroid-induced OHT rodent model
|
Cassidy, Paul S.
|
|
|
20 |
C |
p. 86-94
|
artikel
|
| 57 |
Spatiotemporal in vivo tracking of polyclonal human regulatory T cells (Tregs) reveals a role for innate immune cells in Treg transplant recruitment
|
Jacob, Jacinta
|
|
|
20 |
C |
p. 324-336
|
artikel
|
| 58 |
Systematic improvements in lentiviral transduction of primary human natural killer cells undergoing ex vivo expansion
|
Allan, David S.J.
|
|
|
20 |
C |
p. 559-571
|
artikel
|
| 59 |
Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A
|
Biferi, Maria Grazia
|
|
|
20 |
C |
p. 1-17
|
artikel
|
| 60 |
Targeted epigenetic repression by CRISPR/dSaCas9 suppresses pathogenic DUX4-fl expression in FSHD
|
Himeda, Charis L.
|
|
|
20 |
C |
p. 298-311
|
artikel
|
| 61 |
The microRNA cluster miR-30b/-30d prevents tumor cell switch from an epithelial to a mesenchymal-like phenotype in GBC
|
Cui, Kang
|
|
|
20 |
C |
p. 716-725
|
artikel
|
| 62 |
The TOP vector: a new high-titer lentiviral construct for delivery of sgRNAs and transgenes to primary T cells
|
Humes, Daryl
|
|
|
20 |
C |
p. 30-38
|
artikel
|
| 63 |
Use of 27G needles improves sensitivity and performance of ATCC anaerobe reference microorganism detection in BacT/Alert system
|
Pasqua, Salvatore
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|
|
20 |
C |
p. 542-550
|
artikel
|
| 64 |
UTX/KDM6A deletion promotes the recovery of spinal cord injury by epigenetically triggering intrinsic neural regeneration
|
Guo, Zhu
|
|
|
20 |
C |
p. 337-349
|
artikel
|
| 65 |
Vinblastine treatment decreases the undifferentiated cell contamination of human iPSC-derived intestinal epithelial-like cells
|
Ichikawa, Moe
|
|
|
20 |
C |
p. 463-472
|
artikel
|
| 66 |
Visceral adipose tissue-directed FGF21 gene therapy improves metabolic and immune health in BTBR mice
|
Queen, Nicholas J.
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|
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20 |
C |
p. 409-422
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artikel
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