| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
A cellular disease model toward gene therapy of TGM1-dependent lamellar ichthyosis
|
Sercia, Laura
|
|
|
32 |
3 |
p.
|
artikel
|
| 2 |
A GLB1 transgene with enhanced therapeutic potential for the preclinical development of ex-vivo gene therapy to treat mucopolysaccharidosis type IVB
|
Crippa, Stefania
|
|
|
32 |
3 |
p.
|
artikel
|
| 3 |
An amplification-free CRISPR-Cas12a assay for titer determination and composition analysis of the rAAV genome
|
Yu, Lei
|
|
|
32 |
3 |
p.
|
artikel
|
| 4 |
An autonucleolytic suspension HEK293F host cell line for high-titer serum-free AAV5 and AAV9 production with reduced levels of DNA impurity
|
Howe, Geoffrey
|
|
|
32 |
3 |
p.
|
artikel
|
| 5 |
A novel approach to quantitate biodistribution and transduction of adeno-associated virus gene therapy using radiolabeled AAV vectors in mice
|
Wang, Hongzhi
|
|
|
32 |
3 |
p.
|
artikel
|
| 6 |
A pseudotyped adenovirus serotype 5 vector with serotype 49 fiber knob is an effective vector for vaccine and gene therapy applications
|
Bliss, Carly M.
|
|
|
32 |
3 |
p.
|
artikel
|
| 7 |
A randomized, double-blind phase 2b trial to evaluate efficacy of ChAd63-KH for treatment of post kala-azar dermal leishmaniasis
|
Younis, Brima M.
|
|
|
32 |
3 |
p.
|
artikel
|
| 8 |
A self-complementary AAV proviral plasmid that reduces cross-packaging and ITR promoter activity in AAV vector preparations
|
Taylor, Noah K.
|
|
|
32 |
3 |
p.
|
artikel
|
| 9 |
Assessment of adeno-associated virus purity by capillary electrophoresis-based western
|
Acevedo, Julyana
|
|
|
32 |
3 |
p.
|
artikel
|
| 10 |
Assessment of adeno-associated virus purity by capillary electrophoresis-based western
|
Acevedo, Julyana
|
|
|
32 |
3 |
p.
|
artikel
|
| 11 |
Atelocollagen supports three-dimensional culture of human induced pluripotent stem cells
|
Nakashima, Yoshiki
|
|
|
32 |
3 |
p.
|
artikel
|
| 12 |
Auto-expansion of in vivo HDAd-transduced hematopoietic stem cells by constitutive expression of tHMGA2
|
Wang, Hongjie
|
|
|
32 |
3 |
p.
|
artikel
|
| 13 |
Biolayer interferometry for adeno-associated virus capsid titer measurement and applications to upstream and downstream process development
|
Leibiger, Thomas M.
|
|
|
32 |
3 |
p.
|
artikel
|
| 14 |
Cardiac gene delivery using ultrasound: State of the field
|
Singh, Davindra
|
|
|
32 |
3 |
p.
|
artikel
|
| 15 |
Characterization of AAV vectors: A review of analytical techniques and critical quality attributes
|
Kontogiannis, Theodoros
|
|
|
32 |
3 |
p.
|
artikel
|
| 16 |
Characterization of residual microRNAs in AAV vector batches produced in HEK293 mammalian cells and Sf9 insect cells
|
Penaud-Budloo, Magalie
|
|
|
32 |
3 |
p.
|
artikel
|
| 17 |
Combination AAV therapy with galectin-1 and SOD1 downregulation demonstrates superior therapeutic effect in a severe ALS mouse model
|
Baird, Megan C.
|
|
|
32 |
3 |
p.
|
artikel
|
| 18 |
Consensus-driven target product profiles for curative sickle cell disease gene therapies
|
Bukini, Daima
|
|
|
32 |
3 |
p.
|
artikel
|
| 19 |
CRISPR-Cas9-mediated genome editing delivered by a single AAV9 vector inhibits HSV-1 reactivation in a latent rabbit keratitis model
|
Amrani, Nadia
|
|
|
32 |
3 |
p.
|
artikel
|
| 20 |
DNA-PK inhibition enhances gene editing efficiency in HSPCs for CRISPR-based treatment of X-linked hyper IgM syndrome
|
Pugliano, Cole M.
|
|
|
32 |
3 |
p.
|
artikel
|
| 21 |
Engineering single-cycle MeV vector for CRISPR-Cas9 gene editing
|
Rallabandi, Ramya
|
|
|
32 |
3 |
p.
|
artikel
|
| 22 |
Evaluation of anti-vector immune responses to adenovirus-mediated lung gene therapy and modulation by αCD20
|
Clark, Robert D.E.
|
|
|
32 |
3 |
p.
|
artikel
|
| 23 |
Evaluation of a rapid multi-attribute combinatorial high-throughput UV-Vis/DLS/SLS analytical platform for rAAV quantification and characterization
|
Liu, Xueyuan
|
|
|
32 |
3 |
p.
|
artikel
|
| 24 |
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies
|
Chhabra, Amit
|
|
|
32 |
3 |
p.
|
artikel
|
| 25 |
Highly branched poly β-amino ester/CpG-depleted CFTR plasmid nanoparticles for non-viral gene therapy in lung cystic fibrosis disease
|
Qiu, Bei
|
|
|
32 |
3 |
p.
|
artikel
|
| 26 |
Humoral and cellular immune responses to AAV delivery in the airway
|
Wang, Dongxin
|
|
|
32 |
3 |
p.
|
artikel
|
| 27 |
Lentiviral vector packaging and producer cell lines yield titers equivalent to the industry-standard four-plasmid process
|
Tridgett, Matthew
|
|
|
32 |
3 |
p.
|
artikel
|
| 28 |
Linker-specific monoclonal antibodies present a simple and reliable detection method for scFv-based CAR NK cells
|
Schindler, Katharina
|
|
|
32 |
3 |
p.
|
artikel
|
| 29 |
Lipid nanoparticle-encapsulated DOCK11-siRNA efficiently reduces hepatitis B virus cccDNA level in infected mice
|
Okada, Hikari
|
|
|
32 |
3 |
p.
|
artikel
|
| 30 |
Lipid nanoparticle encapsulation of a Delta spike-CD40L DNA vaccine improves effectiveness against Omicron challenge in Syrian hamsters
|
Tamming, Levi
|
|
|
32 |
3 |
p.
|
artikel
|
| 31 |
Macrophage manufacturing and engineering with 5′-Cap1 and N1-methylpseudouridine-modified mRNA
|
Zhang, Peixuan
|
|
|
32 |
3 |
p.
|
artikel
|
| 32 |
Mesenchymal stromal cells protect combined oncolytic and helper-dependent adenoviruses from humoral immunity
|
Özcan, Ada Irmak
|
|
|
32 |
3 |
p.
|
artikel
|
| 33 |
Modulation of the pharmacokinetics of soluble ACE2 decoy receptors through glycosylation
|
Skeeters, Savanna
|
|
|
32 |
3 |
p.
|
artikel
|
| 34 |
Persistent tailoring of MSC activation through genetic priming
|
Beauregard, Michael A.
|
|
|
32 |
3 |
p.
|
artikel
|
| 35 |
Predictive power of deleterious single amino acid changes to infer on AAV2 and AAV2-13 capsids fitness
|
La Bella, Tiziana
|
|
|
32 |
3 |
p.
|
artikel
|
| 36 |
Probing recombinant AAV capsid integrity and genome release after thermal stress by mass photometry
|
Ebberink, Eduard H.T.M.
|
|
|
32 |
3 |
p.
|
artikel
|
| 37 |
Production of recombinant adeno-associated virus 5 using a novel self-attenuating adenovirus production platform
|
Roach, Matthew K.
|
|
|
32 |
3 |
p.
|
artikel
|
| 38 |
Protein is expressed in all major organs after intravenous infusion of mRNA-lipid nanoparticles in swine
|
Ferraresso, Francesca
|
|
|
32 |
3 |
p.
|
artikel
|
| 39 |
Quantification of circulating TCR-engineered T cells targeting a human endogenous retrovirus post-adoptive transfer using nanoplate digital PCR
|
Barisic, Stefan
|
|
|
32 |
3 |
p.
|
artikel
|
| 40 |
Quantification of full and empty particles of adeno-associated virus vectors via a novel dual fluorescence-linked immunosorbent assay
|
Soth, Sereirath
|
|
|
32 |
3 |
p.
|
artikel
|
| 41 |
Resolving hidden subpopulations of filled AAVs by probing capsid integrity
|
Wu, Weida
|
|
|
32 |
3 |
p.
|
artikel
|
| 42 |
Subacute liver injury in two young infants following gene replacement therapy for spinal muscular atrophy
|
Ables, Cassie
|
|
|
32 |
3 |
p.
|
artikel
|
| 43 |
Suppression of toxic transgene expression by optimized artificial miRNAs increases AAV vector yields in HEK-293 cells
|
Blahetek, Gina
|
|
|
32 |
3 |
p.
|
artikel
|
| 44 |
Systemic delivery of AAV-GCDH ameliorates HLD-induced phenotype in a glutaric aciduria type I mouse model
|
Mateu-Bosch, Anna
|
|
|
32 |
3 |
p.
|
artikel
|
| 45 |
Temporal insights into molecular and cellular responses during rAAV production in HEK293T cells
|
Patra, Alok Tanala
|
|
|
32 |
3 |
p.
|
artikel
|
| 46 |
The longitudinal kinetics of AAV5 vector integration profiles and evaluation of clonal expansion in mice
|
Ismail, Ashrafali Mohamed
|
|
|
32 |
3 |
p.
|
artikel
|
| 47 |
The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome
|
Jeon, Shin
|
|
|
32 |
3 |
p.
|
artikel
|
| 48 |
Toward a large-batch manufacturing process for silicon-stabilized lipid nanoparticles: A highly customizable RNA delivery platform
|
Saffie-Siebert, Suzanne
|
|
|
32 |
3 |
p.
|
artikel
|
| 49 |
5′ Transgenes drive leaky expression of 3′ transgenes in Cre-inducible bi-cistronic vectors
|
Osanai, Yasuyuki
|
|
|
32 |
3 |
p.
|
artikel
|
| 50 |
Treating late-onset Tay Sachs disease: Brain delivery with a dual trojan horse protein
|
Osher, Esther
|
|
|
32 |
3 |
p.
|
artikel
|
| 51 |
Validation of high-sensitivity assays to quantitate cerebrospinal fluid and serum β-galactosidase activity in patients with GM1-gangliosidosis
|
Quadrini, Karen J.
|
|
|
32 |
3 |
p.
|
artikel
|
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