| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAV capsids target muscle-resident cells with different efficiencies—A comparative study between AAV8, AAVMYO, and AAVMYO2
|
McGowan, Timothy J.
|
|
|
33 |
2 |
p.
|
artikel
|
| 2 |
AAV-mediated inner ear gene delivery triggers mild host immune responses in the mammalian inner ear
|
Ishibashi, Yasuko
|
|
|
33 |
2 |
p.
|
artikel
|
| 3 |
Advancing AAV technology: From capsid design to scalable manufacturing
|
Stone, Daniel
|
|
|
33 |
2 |
p.
|
artikel
|
| 4 |
Advancing adeno-associated virus for Duchenne muscular dystrophy treatment: Moving beyond rodent models
|
Ikotun, Oluwatayo F.
|
|
|
33 |
2 |
p.
|
artikel
|
| 5 |
A method to validate viral copy-number assay involving a hybrid amplicon and duplex droplet digital PCR
|
Wu, Raymond
|
|
|
33 |
2 |
p.
|
artikel
|
| 6 |
A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease
|
Sellier, P.
|
|
|
33 |
2 |
p.
|
artikel
|
| 7 |
Anti-HIV-1 HSPC-based gene therapy with safety kill switch to defend against and attack HIV-1 infection
|
Guo, Qi
|
|
|
33 |
2 |
p.
|
artikel
|
| 8 |
Anti-TNF-α antisense-oligonucleotide-conjugated PLG nanoparticles protect transplanted islets
|
Bealer, Elizabeth J.
|
|
|
33 |
2 |
p.
|
artikel
|
| 9 |
A sensitive sample preparation pipeline for adventitious virus detection using Oxford Nanopore sequencing
|
Tsinda, Emmanuel K.
|
|
|
33 |
2 |
p.
|
artikel
|
| 10 |
Assessing the safety of gene therapy vectors expressing an enhanced gamma-globin gene for the cure of sickle cell anemia
|
Rivella, Stefano
|
|
|
33 |
2 |
p.
|
artikel
|
| 11 |
A two-pass anion-exchange chromatography strategy for enrichment of full capsids in manufacturing of adeno-associated viral vectors
|
Thakur, Garima
|
|
|
33 |
2 |
p.
|
artikel
|
| 12 |
Bystander editing by adenine base editors impairs vision restoration in a mouse model of Leber congenital amaurosis
|
Lee, Seok-Hoon
|
|
|
33 |
2 |
p.
|
artikel
|
| 13 |
Characterization of factors that influence rAAV yield and quality when produced using rHSV co-infection
|
E, Xiaofei
|
|
|
33 |
2 |
p.
|
artikel
|
| 14 |
Characterization of intact mRNA-based therapeutics by charge detection mass spectrometry and mass photometry
|
Deslignière, Evolène
|
|
|
33 |
2 |
p.
|
artikel
|
| 15 |
Common AAV gene therapy vectors show nonselective transduction of ex vivo human brain tissue
|
McGinnis, JP
|
|
|
33 |
2 |
p.
|
artikel
|
| 16 |
Comparison of NSG-Quad and MISTRG-6 humanized mice for modeling circulating and tumor-infiltrating human myeloid cells
|
Chen, Anna
|
|
|
33 |
2 |
p.
|
artikel
|
| 17 |
Development of circular AAV cargos for targeted seamless insertion with large serine integrases
|
Estes, Brett J.G.
|
|
|
33 |
2 |
p.
|
artikel
|
| 18 |
Efficacy and preliminary safety assessment of EXG001-307 AAV gene therapy for spinal muscular atrophy
|
Song, Chunjuan
|
|
|
33 |
2 |
p.
|
artikel
|
| 19 |
Engineered ACE2 decoy in dry powder form for inhalation: A novel therapy for SARS-CoV-2 variants
|
Ito, Takaaki
|
|
|
33 |
2 |
p.
|
artikel
|
| 20 |
Enhanced retinal pigment epithelial cells as a delivery vehicle for retinal disease
|
Reddy, Avril
|
|
|
33 |
2 |
p.
|
artikel
|
| 21 |
Enhancing titers of therapeutic lentiviral vectors using PKC agonists
|
Moore-Kelly, Charles
|
|
|
33 |
2 |
p.
|
artikel
|
| 22 |
Evaluation of AAV vectors with tissue-specific or ubiquitous promoters in a mouse model of mucopolysaccharidosis type IVA
|
Khan, Shaukat A.
|
|
|
33 |
2 |
p.
|
artikel
|
| 23 |
Evaluation of the loading capacity and patterns of packaged DNA in AAV genomes of different sizes using long-read sequencing
|
Kosaka, Mitsuki
|
|
|
33 |
2 |
p.
|
artikel
|
| 24 |
Exploring human plasma proteomic variations in mucolipidosis type IV
|
Tobin, Brendan R.
|
|
|
33 |
2 |
p.
|
artikel
|
| 25 |
Exploring the potential of saponins as adjuvants in lipid-nanoparticle-based mRNA vaccines
|
Eygeris, Yulia
|
|
|
33 |
2 |
p.
|
artikel
|
| 26 |
Harnessing synaptic vesicle release and recycling with antibody shuttle for targeted delivery of therapeutics to neurons
|
Yee, Karen Kar Lye
|
|
|
33 |
2 |
p.
|
artikel
|
| 27 |
Impact of DNase digestion on titer measurements of engineered adeno-associated virus serotypes
|
Zin, Emilia A.
|
|
|
33 |
2 |
p.
|
artikel
|
| 28 |
Improving resolution with single-cell detection of CRISPR off-target events
|
Lee, Ciaran M.
|
|
|
33 |
2 |
p.
|
artikel
|
| 29 |
Interlaboratory assessment of candidate reference materials for lentiviral vector copy number and integration site measurements
|
He, Hua-Jun
|
|
|
33 |
2 |
p.
|
artikel
|
| 30 |
In vivo HSC transduction in humanized mice mediated by novel capsid-modified HDAd vectors
|
Georgakopoulou, Aphrodite
|
|
|
33 |
2 |
p.
|
artikel
|
| 31 |
In-vivo-targeted gene delivery using adenovirus-antibody site-specific covalent conjugates
|
Rice-Boucher, Paul J.
|
|
|
33 |
2 |
p.
|
artikel
|
| 32 |
Ionizable lipid nanoparticles with functionalized PEG-lipids increase retention in the tumor microenvironment
|
Jester, Matthew
|
|
|
33 |
2 |
p.
|
artikel
|
| 33 |
iPSC-hepatocyte organoids as a novel platform to predict AAV gene therapy efficacy
|
Berreur, Estelle
|
|
|
33 |
2 |
p.
|
artikel
|
| 34 |
Lipid nanoparticles: Composition, formulation, and application
|
Xu, Sijia
|
|
|
33 |
2 |
p.
|
artikel
|
| 35 |
Lipid nanoparticles from L. meyenii Walp mitigate sepsis through multimodal protein corona formation
|
Sung, Junsik J.
|
|
|
33 |
2 |
p.
|
artikel
|
| 36 |
Mapping administration route-dependent transduction profiles of commonly used AAV variants in mice by barcode amplicon sequencing
|
Abele, Sarah
|
|
|
33 |
2 |
p.
|
artikel
|
| 37 |
Methods for cell- and tissue-specific DNA aptamer selection
|
Wilbanks, Brandon A.
|
|
|
33 |
2 |
p.
|
artikel
|
| 38 |
Optimization of hypo-alloimmunogenic multispecific CAR-T and SARS-CoV-2-specific T cells for off-the-shelf adoptive cell therapy
|
Ng, King Pan
|
|
|
33 |
2 |
p.
|
artikel
|
| 39 |
Optimization of lentiviral delivery of barcoded anti-CD20 chimeric antigen receptors into rhesus macaque and human natural killer cells
|
Hayal, Taha B.
|
|
|
33 |
2 |
p.
|
artikel
|
| 40 |
Precise measurement of CRISPR genome editing outcomes through single-cell DNA sequencing
|
Kalter, Nechama
|
|
|
33 |
2 |
p.
|
artikel
|
| 41 |
Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models
|
Shrestha, Archana
|
|
|
33 |
2 |
p.
|
artikel
|
| 42 |
Preclinical studies of an AAV8-CYP4V2 gene therapy VGR-R01 for the treatment of Bietti crystalline dystrophy
|
Luo, Wenjing
|
|
|
33 |
2 |
p.
|
artikel
|
| 43 |
Preclinical toxicological assessment of an α-galactosylceramide-adjuvanted mRNA cancer vaccine in Wistar Han rats and domestic pigs
|
Meulewaeter, Sofie
|
|
|
33 |
2 |
p.
|
artikel
|
| 44 |
Redosing of AAV vectors in the brain after intracranial injections: Low-ish hanging fruit?
|
Maguire, Casey A.
|
|
|
33 |
2 |
p.
|
artikel
|
| 45 |
Self-silencing adenovirus enables precise infectious titration of recombinant adeno-associated viral vectors
|
Fustinoni, Chloé
|
|
|
33 |
2 |
p.
|
artikel
|
| 46 |
Serum factors create species-specific barriers to hepatic gene transfer by lipid nanoparticles in liver-humanized mice
|
Stone, Daniel
|
|
|
33 |
2 |
p.
|
artikel
|
| 47 |
Species-specific gene expression manipulation in humanized livers of chimeric mice via siRNA-encapsulated lipid nanoparticle treatment
|
Yamazaki, Kazuto
|
|
|
33 |
2 |
p.
|
artikel
|
| 48 |
Targeted spiral ganglion neuron degeneration in parvalbumin-Cre neonatal mice
|
Nguyen, Nhi V.
|
|
|
33 |
2 |
p.
|
artikel
|
| 49 |
Targeting the trunk of multi-root common epilepsy with gene therapy
|
Sheibani, Mehrnaz
|
|
|
33 |
2 |
p.
|
artikel
|
| 50 |
The clinical quality management system of advanced therapy medicinal products in the hospital setting: A scoping review
|
Shi, Junnan
|
|
|
33 |
2 |
p.
|
artikel
|
| 51 |
The manufacture of AAV for gene therapy applications using a closed, semi-automated hollow-fiber bioreactor
|
Soula, Adrien
|
|
|
33 |
2 |
p.
|
artikel
|
| 52 |
The RNA content of extracellular vesicles from gene-edited PRPF31 +/− hiPSC-RPE show potential as biomarkers of retinal degeneration
|
Getachew, Heran
|
|
|
33 |
2 |
p.
|
artikel
|
| 53 |
Unfolding of viral protein 1 N-termini facilitates genome ejection from recombinant adeno-associated virus serotype 8
|
Yamaguchi, Yuki
|
|
|
33 |
2 |
p.
|
artikel
|
| 54 |
Virally mediated enhancement of efferent inhibition reduces acoustic trauma in wild-type murine cochleas
|
Slika, Eleftheria
|
|
|
33 |
2 |
p.
|
artikel
|
| 55 |
Widespread tissue delivery of antagomiRs via intramuscular administration
|
Messios, Christodoulos
|
|
|
33 |
2 |
p.
|
artikel
|
| 56 |
WRAP-based nanoparticles for siRNA delivery in zebrafish embryos by simple bath immersion
|
Konate, Karidia
|
|
|
33 |
2 |
p.
|
artikel
|
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