| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study
|
Hu, Huimin
|
|
|
23 |
C |
p. 644-658
|
artikel
|
| 2 |
AAV-mediated PEX1 gene augmentation improves visual function in the PEX1-Gly844Asp mouse model for mild Zellweger spectrum disorder
|
Argyriou, Catherine
|
|
|
23 |
C |
p. 225-240
|
artikel
|
| 3 |
AAV vectors accumulate in the pineal gland after injections into the brain or spinal cord
|
Steward, Oswald
|
|
|
23 |
C |
p. 406-417
|
artikel
|
| 4 |
A chimeric virus-based probe unambiguously detects live circulating tumor cells with high specificity and sensitivity
|
Fu, Xinping
|
|
|
23 |
C |
p. 78-86
|
artikel
|
| 5 |
Adeno-associated viral vector serotype 9-based gene replacement therapy for SURF1-related Leigh syndrome
|
Ling, Qinglan
|
|
|
23 |
C |
p. 158-168
|
artikel
|
| 6 |
Biodistribution of intravitreal lenadogene nolparvovec gene therapy in nonhuman primates
|
Calkins, David J.
|
|
|
23 |
C |
p. 307-318
|
artikel
|
| 7 |
Characterization of a library of 20 HBV-specific MHC class II-restricted T cell receptors
|
Schreiber, Sophia
|
|
|
23 |
C |
p. 476-489
|
artikel
|
| 8 |
Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette
|
Kumar, Sandeep R.P.
|
|
|
23 |
C |
p. 98-107
|
artikel
|
| 9 |
Comparison of analytical techniques to quantitate the capsid content of adeno-associated viral vectors
|
Werle, Amanda K.
|
|
|
23 |
C |
p. 254-262
|
artikel
|
| 10 |
Complete intra-laboratory validation of a LAL assay for bacterial endotoxin determination in EBV-specific cytotoxic T lymphocytes
|
Pasqua, Salvatore
|
|
|
23 |
C |
p. 567-568
|
artikel
|
| 11 |
CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease
|
Demirci, Selami
|
|
|
23 |
C |
p. 276-285
|
artikel
|
| 12 |
Current landscape of clinical development and approval of advanced therapies
|
Iglesias-Lopez, Carolina
|
|
|
23 |
C |
p. 606-618
|
artikel
|
| 13 |
Defining the optimal dose and therapeutic window in SMA with respiratory distress type I model mice, FVB/NJ-Ighmpb2 nmd-2J
|
Shababi, Monir
|
|
|
23 |
C |
p. 23-32
|
artikel
|
| 14 |
Development of a one-step RT-ddPCR method to determine the expression and potency of AAV vectors
|
Clarner, Pete
|
|
|
23 |
C |
p. 68-77
|
artikel
|
| 15 |
Digital PCR to quantify ChAdOx1 nCoV-19 copies in blood and tissues
|
Badbaran, Anita
|
|
|
23 |
C |
p. 418-423
|
artikel
|
| 16 |
Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device
|
Olgasi, Cristina
|
|
|
23 |
C |
p. 551-566
|
artikel
|
| 17 |
Engineered mesenchymal stromal cell therapy during human lung ex vivo lung perfusion is compromised by acidic lung microenvironment
|
Nykänen, Antti I.
|
|
|
23 |
C |
p. 184-197
|
artikel
|
| 18 |
Enzymatically produced piggyBac transposon vectors for efficient non-viral manufacturing of CD19-specific CAR T cells
|
Kaštánková, Iva
|
|
|
23 |
C |
p. 119-127
|
artikel
|
| 19 |
Evaluation of two in vitro assays for tumorigenicity assessment of CRISPR-Cas9 genome-edited cells
|
Lemmens, Myriam
|
|
|
23 |
C |
p. 241-253
|
artikel
|
| 20 |
Genetically edited hepatic cells expressing the NTCP-S267F variant are resistant to hepatitis B virus infection
|
Uchida, Takuro
|
|
|
23 |
C |
p. 597-605
|
artikel
|
| 21 |
GJB2 gene therapy and conditional deletion reveal developmental stage-dependent effects on inner ear structure and function
|
Guo, Jingying
|
|
|
23 |
C |
p. 319-333
|
artikel
|
| 22 |
High throughput screening of novel AAV capsids identifies variants for transduction of adult NSCs within the subventricular zone
|
Kremer, Lukas P.M.
|
|
|
23 |
C |
p. 33-50
|
artikel
|
| 23 |
Immunocompromised Cas9 transgenic mice for rapid in vivo assessment of host factors involved in highly pathogenic virus infection
|
Collette, Nicole
|
|
|
23 |
C |
p. 286-295
|
artikel
|
| 24 |
Immunological evaluation of an inactivated SARS-CoV-2 vaccine in rhesus macaques
|
Chen, Hongbo
|
|
|
23 |
C |
p. 108-118
|
artikel
|
| 25 |
Implications of circulating neurofilaments for spinal muscular atrophy treatment early in life: A case series
|
Alves, Christiano R.R.
|
|
|
23 |
C |
p. 524-538
|
artikel
|
| 26 |
Improved functionality and potency of next generation BinMLV viral vectors toward safer gene therapy
|
Van Looveren, Dominique
|
|
|
23 |
C |
p. 51-67
|
artikel
|
| 27 |
Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina
|
Tornabene, Patrizia
|
|
|
23 |
C |
p. 448-459
|
artikel
|
| 28 |
In vivo genome editing at the albumin locus to treat methylmalonic acidemia
|
Schneller, Jessica L.
|
|
|
23 |
C |
p. 619-632
|
artikel
|
| 29 |
Lentiviral and adeno-associated vectors efficiently transduce mouse T lymphocytes when targeted to murine CD8
|
Michels, Alexander
|
|
|
23 |
C |
p. 334-347
|
artikel
|
| 30 |
Minimal impact of ZAP on lentiviral vector production and transduction efficiency
|
Sertkaya, Helin
|
|
|
23 |
C |
p. 147-157
|
artikel
|
| 31 |
miR-223-3p and miR-24-3p as novel serum-based biomarkers for myotonic dystrophy type 1
|
Koutalianos, Demetris
|
|
|
23 |
C |
p. 169-183
|
artikel
|
| 32 |
Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells
|
Arjomandnejad, Motahareh
|
|
|
23 |
C |
p. 490-506
|
artikel
|
| 33 |
Monitoring CAR T cell generation with a CD8-targeted lentiviral vector by single-cell transcriptomics
|
Charitidis, Filippos T.
|
|
|
23 |
C |
p. 359-369
|
artikel
|
| 34 |
Multiplex CRISPR/Cas9 genome editing in hematopoietic stem cells for fetal hemoglobin reinduction generates chromosomal translocations
|
Samuelson, Clare
|
|
|
23 |
C |
p. 507-523
|
artikel
|
| 35 |
Nonconditioned ADA-SCID gene therapy reveals ADA requirement in the hematopoietic system and clonal dominance of vector-marked clones
|
Uchiyama, Toru
|
|
|
23 |
C |
p. 424-433
|
artikel
|
| 36 |
Novel TCR-like CAR-T cells targeting an HLA∗0201-restricted SSX2 epitope display strong activity against acute myeloid leukemia
|
Raskin, Scott
|
|
|
23 |
C |
p. 296-306
|
artikel
|
| 37 |
Optimization of AAV6 transduction enhances site-specific genome editing of primary human lymphocytes
|
Rogers, Geoffrey L.
|
|
|
23 |
C |
p. 198-209
|
artikel
|
| 38 |
Optimized pharmacological control over the AAV-Gene-Switch vector for regulable gene therapy
|
Cheng, Shi
|
|
|
23 |
C |
p. 1-10
|
artikel
|
| 39 |
Optimizing AAV2/6 microglial targeting identified enhanced efficiency in the photoreceptor degenerative environment
|
Maes, Margaret E.
|
|
|
23 |
C |
p. 210-224
|
artikel
|
| 40 |
Peripheral leukemia burden at time of apheresis negatively affects the clinical efficacy of CART19 in refractory or relapsed B-ALL
|
Deng, Biping
|
|
|
23 |
C |
p. 633-643
|
artikel
|
| 41 |
piggyBac system to co-express NKG2D CAR and IL-15 to augment the in vivo persistence and anti-AML activity of human peripheral blood NK cells
|
Du, Zhicheng
|
|
|
23 |
C |
p. 582-596
|
artikel
|
| 42 |
Production and purification of high-titer OrfV for preclinical studies in vaccinology and cancer therapy
|
van Vloten, Jacob P.
|
|
|
23 |
C |
p. 434-447
|
artikel
|
| 43 |
Quantification of cell-free DNA for the analysis of CD19-CAR-T cells during lymphoma treatment
|
Mika, Thomas
|
|
|
23 |
C |
p. 539-550
|
artikel
|
| 44 |
Quantitative analysis of genome packaging in recombinant AAV vectors by charge detection mass spectrometry
|
Barnes, Lauren F.
|
|
|
23 |
C |
p. 87-97
|
artikel
|
| 45 |
Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis
|
Taghian, Toloo
|
|
|
23 |
C |
p. 128-134
|
artikel
|
| 46 |
Safe and stable generation of induced pluripotent stem cells using doggybone DNA vectors
|
Thornton, Christopher D.
|
|
|
23 |
C |
p. 348-358
|
artikel
|
| 47 |
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys
|
Baruteau, Julien
|
|
|
23 |
C |
p. 135-146
|
artikel
|
| 48 |
Safety and efficiency modifications of SIV-based integrase-defective lentiviral vectors for immunization
|
Bona, Roberta
|
|
|
23 |
C |
p. 263-275
|
artikel
|
| 49 |
SAG therapy restores bone growth and reduces enchondroma incidence in a model of skeletal chondrodysplasias caused by Ihh deficiency
|
Li, Xinhua
|
|
|
23 |
C |
p. 461-475
|
artikel
|
| 50 |
Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal
|
Marcó, Sara
|
|
|
23 |
C |
p. 370-389
|
artikel
|
| 51 |
Specific gene expression in unmyelinated dorsal root ganglion neurons in nonhuman primates by intra-nerve injection of AAV 6 vector
|
Kudo, Moeko
|
|
|
23 |
C |
p. 11-22
|
artikel
|
| 52 |
Tips and tricks for successfully culturing and adapting human induced pluripotent stem cells
|
Castro-Viñuelas, Rocío
|
|
|
23 |
C |
p. 569-581
|
artikel
|
| 53 |
Transgene distribution and immune response after ultrasound delivery of rAAV9 and PHP.B to the brain in a mouse model of amyloidosis
|
Kofoed, Rikke Hahn
|
|
|
23 |
C |
p. 390-405
|
artikel
|
| 54 |
Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice
|
Hamm, Shelby E.
|
|
|
23 |
C |
p. 460
|
artikel
|
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