| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAVolve: Concatenated long-read deep sequencing enables whole capsid tracking during shuffled AAV library selection
|
Scott, Suzanne
|
|
|
32 |
4 |
p.
|
artikel
|
| 2 |
Adeno-associated virus serotype 9 antibodies in neonates and young children: Seroprevalence and kinetics
|
van Olden, Rudolf W.
|
|
|
32 |
4 |
p.
|
artikel
|
| 3 |
Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders
|
Zhou, Livia
|
|
|
32 |
4 |
p.
|
artikel
|
| 4 |
An HPLC-SEC-based rapid quantification method for vesicular stomatitis virus particles to facilitate process development
|
Schimek, Adrian
|
|
|
32 |
4 |
p.
|
artikel
|
| 5 |
An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs
|
Vasireddy, Vidyullatha
|
|
|
32 |
4 |
p.
|
artikel
|
| 6 |
A novel AAV9-dual microRNA-vector targeting GRIK2 in the hippocampus as a treatment for mesial temporal lobe epilepsy
|
Baudouin, Stéphane J.
|
|
|
32 |
4 |
p.
|
artikel
|
| 7 |
CAR-NK cells derived from cord blood originate mainly from CD56−CD7+CD34−HLA-DR−Lin− NK progenitor cells
|
Wibowo, Tansri
|
|
|
32 |
4 |
p.
|
artikel
|
| 8 |
Characterization of drusen formation in a primary porcine tissue culture model of dry AMD
|
Shaw, Erika M.
|
|
|
32 |
4 |
p.
|
artikel
|
| 9 |
Characterization of the function of Adenovirus L4 gene products and their impact on AAV vector production
|
Nie, Yingchao
|
|
|
32 |
4 |
p.
|
artikel
|
| 10 |
Comparative assessment of the transduction efficiency and safety associated with the delivery of AAV9-GFP vector via lumbar puncture to cynomolgus macaques with and without anti-AAV9 pre-existing antibodies
|
Guibinga, Ghiabe H.
|
|
|
32 |
4 |
p.
|
artikel
|
| 11 |
Comparison and cross-validation of long-read and short-read target-enrichment sequencing methods to assess AAV vector integration into host genome
|
Sheehan, Mark
|
|
|
32 |
4 |
p.
|
artikel
|
| 12 |
Comprehensive analysis of off-target and on-target effects resulting from liver-directed CRISPR-Cas9-mediated gene targeting with AAV vectors
|
Singh, Kshitiz
|
|
|
32 |
4 |
p.
|
artikel
|
| 13 |
Design and validation of cell-based potency assays for frataxin supplementation treatments
|
Mukherjee, Shibani
|
|
|
32 |
4 |
p.
|
artikel
|
| 14 |
Determining recombinant AAV capsid extracellular and intracellular biodistribution by dual radioisotope labeling
|
Chandler, Randy J.
|
|
|
32 |
4 |
p.
|
artikel
|
| 15 |
DNA contamination within recombinant adeno-associated virus preparations correlates with decreased CD34+ cell clonogenic potential
|
Luthers, Christopher R.
|
|
|
32 |
4 |
p.
|
artikel
|
| 16 |
E2A, VA RNA I, and L4-22k adenoviral helper genes are sufficient for AAV production in HEK293 cells
|
Doshi, Jiten
|
|
|
32 |
4 |
p.
|
artikel
|
| 17 |
Efficient generation of liver sinusoidal endothelial-like cells secreting coagulation factor VIII from human induced pluripotent stem cells
|
Mitani, Seiji
|
|
|
32 |
4 |
p.
|
artikel
|
| 18 |
Exogenous expression of ATP8, a mitochondrial encoded protein, from the nucleus in vivo
|
Begelman, David V.
|
|
|
32 |
4 |
p.
|
artikel
|
| 19 |
αFAP-specific nanobodies mediate a highly precise retargeting of modified AAV2 capsids thereby enabling specific transduction of tumor tissues
|
Olarewaju, Olaniyi
|
|
|
32 |
4 |
p.
|
artikel
|
| 20 |
FastAd: A versatile toolkit for rapid generation of single adenoviruses or diverse adenoviral vector libraries
|
Lu, Shao-Chia
|
|
|
32 |
4 |
p.
|
artikel
|
| 21 |
Generation and maintenance of kidney and kidney cancer organoids from patient-derived material for drug development and precision oncology
|
Gubala, Jakub
|
|
|
32 |
4 |
p.
|
artikel
|
| 22 |
Gene therapy and kidney diseases
|
Tavakolidakhrabadi, Nadia
|
|
|
32 |
4 |
p.
|
artikel
|
| 23 |
High-affinity chimeric antigen receptor signaling induces an inflammatory program in human regulatory T cells
|
Cochrane, Russell W.
|
|
|
32 |
4 |
p.
|
artikel
|
| 24 |
Human iPSC-derived neural stem cells engraft and improve pathophysiology of MPS I mice
|
Calhoun, Caitlin C.
|
|
|
32 |
4 |
p.
|
artikel
|
| 25 |
Identification of a novel neutralization epitope in rhesus AAVs
|
Dagotto, Gabriel
|
|
|
32 |
4 |
p.
|
artikel
|
| 26 |
Implementing a robust platform analytical procedure for measuring adeno-associated virus vector genome titer
|
Powers, Thomas W.
|
|
|
32 |
4 |
p.
|
artikel
|
| 27 |
Insights in AAV-mediated antigen-specific immunity and a strategy for AAV vaccine dose reduction through AAV-extracellular vesicle association
|
Molina, Ester
|
|
|
32 |
4 |
p.
|
artikel
|
| 28 |
Insights into post-marketing clinical validation of companion diagnostics with reference to the FDA, EMA, PMDA, and MFDS
|
Kang, Su Lim
|
|
|
32 |
4 |
p.
|
artikel
|
| 29 |
Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I
|
Belur, Lalitha R.
|
|
|
32 |
4 |
p.
|
artikel
|
| 30 |
In vitro and in vivo expansion of CD33/HBG promoter-edited HSPCs with Mylotarg
|
Georgakopoulou, Aphrodite
|
|
|
32 |
4 |
p.
|
artikel
|
| 31 |
Large and small extracellular vesicles from Wharton’s jelly MSCs: Biophysics, function, and strategies to improve immunomodulation
|
Buitrago, July Constanza
|
|
|
32 |
4 |
p.
|
artikel
|
| 32 |
Lentiviral vectors for precise expression to treat X-linked lymphoproliferative disease
|
Ayoub, Paul G.
|
|
|
32 |
4 |
p.
|
artikel
|
| 33 |
Modulation of AAV transduction and integration targeting by topoisomerase poisons
|
Kasimsetty, Aradhana
|
|
|
32 |
4 |
p.
|
artikel
|
| 34 |
mRNA vaccines encoding membrane-anchored RBDs of SARS-CoV-2 mutants induce strong humoral responses and can overcome immune imprinting
|
Al-Wassiti, Hareth A.
|
|
|
32 |
4 |
p.
|
artikel
|
| 35 |
Non-encapsidated miRNA contaminants found in AAV preparations
|
Brimble, Mark A.
|
|
|
32 |
4 |
p.
|
artikel
|
| 36 |
Ocular toxicity, distribution, and shedding of intravitreal AAV-eqIL-10 in horses
|
Young, Kim
|
|
|
32 |
4 |
p.
|
artikel
|
| 37 |
Off-the-shelf allogeneic natural killer cells for the treatment of COVID-19
|
Liu, Winnie L.
|
|
|
32 |
4 |
p.
|
artikel
|
| 38 |
Optimal different adeno-associated virus capsid/promoter combinations to target specific cell types in the common marmoset cerebral cortex
|
Matsuzaki, Yasunori
|
|
|
32 |
4 |
p.
|
artikel
|
| 39 |
Optimizing regulatory frameworks for gene therapies in rare diseases: Challenges and solutions
|
Berry, Diane
|
|
|
32 |
4 |
p.
|
artikel
|
| 40 |
Patient-specific responses to SMN2 splice-modifying treatments in spinal muscular atrophy fibroblasts
|
Signoria, Ilaria
|
|
|
32 |
4 |
p.
|
artikel
|
| 41 |
Physicochemical and biological impacts of light stress on adeno-associated virus serotype 6
|
Takino, Rie
|
|
|
32 |
4 |
p.
|
artikel
|
| 42 |
Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease
|
Liefhebber, Jolanda M.P.
|
|
|
32 |
4 |
p.
|
artikel
|
| 43 |
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration
|
Shim, Juwon
|
|
|
32 |
4 |
p.
|
artikel
|
| 44 |
Progress toward the challenging goal of HDR-based gene editing for hyper-IgM syndrome
|
Bazzo Catto, Luiz Fernando
|
|
|
32 |
4 |
p.
|
artikel
|
| 45 |
Quantitative proteomic analysis of residual host cell protein retention across adeno-associated virus affinity chromatography
|
Leibiger, Thomas M.
|
|
|
32 |
4 |
p.
|
artikel
|
| 46 |
Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I
|
Shi, Xiaoxia
|
|
|
32 |
4 |
p.
|
artikel
|
| 47 |
Single cell and TCR analysis of immune cells from AAV gene therapy-dosed Duchenne muscular dystrophy patients
|
Emami, Michael R.
|
|
|
32 |
4 |
p.
|
artikel
|
| 48 |
Size-exclusion chromatography as a multi-attribute method for process and product characterization of adeno-associated virus
|
Mulagapati, Sri Hari Raju
|
|
|
32 |
4 |
p.
|
artikel
|
| 49 |
Structural characterization and epitope mapping of the AAVX affinity purification ligand
|
Mietzsch, Mario
|
|
|
32 |
4 |
p.
|
artikel
|
| 50 |
Synergy between Lactobacillus murinus and anti-PcrV antibody delivered in the airways to boost protection against Pseudomonas aeruginosa
|
Sécher, Thomas
|
|
|
32 |
4 |
p.
|
artikel
|
| 51 |
Thank you to our 2024 reviewers
|
|
|
|
32 |
4 |
p.
|
artikel
|
| 52 |
Toward a translational gene therapy for mucolipidosis IV
|
Snyder, Brina
|
|
|
32 |
4 |
p.
|
artikel
|
| 53 |
Transcriptomics-informed pharmacology identifies epigenetic and cell cycle regulators that enhance AAV production
|
Tworig, Joshua
|
|
|
32 |
4 |
p.
|
artikel
|
| 54 |
Unlocking DOE potential by selecting the most appropriate design for rAAV optimization
|
Tzimou, Konstantina
|
|
|
32 |
4 |
p.
|
artikel
|
| 55 |
What’s in a word? Defining “gene therapy medicines”
|
von Fritschen, Maren
|
|
|
32 |
4 |
p.
|
artikel
|
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