| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAV-vectored base editor trans-splicing delivers dystrophin repair
|
Li, Zhen
|
|
|
32 |
C |
p. 900-902
|
artikel
|
| 2 |
A conditional RNA Pol II mono-promoter drives HIV-inducible, CRISPR-mediated cyclin T1 suppression and HIV inhibition
|
Chinnapaiyan, Srinivasan
|
|
|
32 |
C |
p. 553-565
|
artikel
|
| 3 |
All roads lead to cure: Diversity of oligonucleotides in DM1 therapy
|
Fiszer, Agnieszka
|
|
|
32 |
C |
p. 898-899
|
artikel
|
| 4 |
A protein domain-oriented approach to expand the opportunities of therapeutic exon skipping for USH2A-associated retinitis pigmentosa
|
Schellens, Renske T.W.
|
|
|
32 |
C |
p. 980-994
|
artikel
|
| 5 |
A review from biological mapping to computation-based subcellular localization
|
Li, Jing
|
|
|
32 |
C |
p. 507-521
|
artikel
|
| 6 |
Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1
|
El Boujnouni, Najoua
|
|
|
32 |
C |
p. 622-636
|
artikel
|
| 7 |
Can the RBD mutation R346X provide an additional fitness to the “variant soup,” including offspring of BQ and XBB of SARS-CoV-2 Omicron for the antibody resistance?
|
Chatterjee, Srijan
|
|
|
32 |
C |
p. 61-63
|
artikel
|
| 8 |
Cellular uptake of modified mRNA occurs via caveolae-mediated endocytosis, yielding high protein expression in slow-dividing cells
|
Del Toro Runzer, Claudia
|
|
|
32 |
C |
p. 960-979
|
artikel
|
| 9 |
Circulating ECVs prevent neurodegeneration and preserve neuronal function in a model of preclinical intracerebral hemorrhage
|
Burkhart, Annette
|
|
|
32 |
C |
p. 668-670
|
artikel
|
| 10 |
Circulating extracellular vesicles promote recovery in a preclinical model of intracerebral hemorrhage
|
Laso-García, Fernando
|
|
|
32 |
C |
p. 247-262
|
artikel
|
| 11 |
Combination of protein and cell internalization SELEX identifies a potential RNA therapeutic and delivery platform to treat EphA2-expressing tumors
|
Santana-Viera, Laura
|
|
|
32 |
C |
p. 758-772
|
artikel
|
| 12 |
CRISPR-Cas9 in hiPSCs: A new era in personalized treatment for Stargardt disease
|
Kuntam, Soujanya
|
|
|
32 |
C |
p. 896-897
|
artikel
|
| 13 |
Deficiency of miR-409-3p improves myocardial neovascularization and function through modulation of DNAJB9/p38 MAPK signaling
|
Bestepe, Furkan
|
|
|
32 |
C |
p. 995-1009
|
artikel
|
| 14 |
Delivery of anti-microRNA-21 by lung-targeted liposomes for pulmonary fibrosis treatment
|
Yan, Lingyue
|
|
|
32 |
C |
p. 36-47
|
artikel
|
| 15 |
Development of DNA aptamers for visualization of glial brain tumors and detection of circulating tumor cells
|
Kichkailo, Anna S.
|
|
|
32 |
C |
p. 267-288
|
artikel
|
| 16 |
Development of new non-viral systems for genetic modification of senescent cells
|
López-Seijas, Junquera
|
|
|
32 |
C |
p. 302-317
|
artikel
|
| 17 |
Differential effects of SARM1 inhibition in traumatic glaucoma and EAE optic neuropathies
|
Liu, Pingting
|
|
|
32 |
C |
p. 13-27
|
artikel
|
| 18 |
Distinct regulatory functions and biological roles of lncRNA splice variants
|
Khan, Muhammad Riaz
|
|
|
32 |
C |
p. 127-143
|
artikel
|
| 19 |
DNAJB6 isoform specific knockdown: Therapeutic potential for limb girdle muscular dystrophy D1
|
Findlay, Andrew R.
|
|
|
32 |
C |
p. 937-948
|
artikel
|
| 20 |
704/DNA vaccines leverage cytoplasmic DNA stimulation to promote anti-HIV neutralizing antibody production in mice and strong immune response against alpha-fetoprotein in non-human primates
|
Colombani, Thibault
|
|
|
32 |
C |
p. 743-757
|
artikel
|
| 21 |
Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies
|
Venkatesan, Vigneshwaran
|
|
|
32 |
C |
p. 671-688
|
artikel
|
| 22 |
Efficient correction of ABCA4 variants by CRISPR-Cas9 in hiPSCs derived from Stargardt disease patients
|
Siles, Laura
|
|
|
32 |
C |
p. 64-79
|
artikel
|
| 23 |
Efficient transfected liposomes co-loaded with pNrf2 and pirfenidone improves safe delivery for enhanced pulmonary fibrosis reversion
|
Chang, Xin
|
|
|
32 |
C |
p. 415-431
|
artikel
|
| 24 |
Expanding DdCBE-mediated targeting scope to aC motif preference in rat
|
Qi, Xiaolong
|
|
|
32 |
C |
p. 1-12
|
artikel
|
| 25 |
Extracellular vesicle-based biovectors in chronic wound healing: Biogenesis and delivery approaches
|
Garima,
|
|
|
32 |
C |
p. 822-840
|
artikel
|
| 26 |
Extracellular vesicle microRNA and protein cargo profiling in three clinical-grade stem cell products reveals key functional pathways
|
Vaka, Ramana
|
|
|
32 |
C |
p. 80-93
|
artikel
|
| 27 |
GOLGA8 increases bulk antisense oligonucleotide uptake and activity in mammalian cells
|
McMahon, Moira A.
|
|
|
32 |
C |
p. 289-301
|
artikel
|
| 28 |
GraphCPIs: A novel graph-based computational model for potential compound-protein interactions
|
Chen, Zhan-Heng
|
|
|
32 |
C |
p. 721-728
|
artikel
|
| 29 |
Identification of shared characteristics in tumor-infiltrating T cells across 15 cancers
|
Jin, Xiyun
|
|
|
32 |
C |
p. 189-202
|
artikel
|
| 30 |
Improved gene therapy for MFRP deficiency-mediated retinal degeneration by knocking down endogenous bicistronic Mfrp and Ctrp5 transcript
|
Tian, Xiao
|
|
|
32 |
C |
p. 843-856
|
artikel
|
| 31 |
Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors
|
Williams, James A.
|
|
|
32 |
C |
p. 494-503
|
artikel
|
| 32 |
Inhaled aptamer therapy: A hopeful therapy for lung disease
|
Martin, M. Elena
|
|
|
32 |
C |
p. 875-876
|
artikel
|
| 33 |
Inhibition of adenovirus replication by CRISPR-Cas9-mediated targeting of the viral E1A gene
|
Didara, Zrinka
|
|
|
32 |
C |
p. 48-60
|
artikel
|
| 34 |
In utero delivery of miRNA induces epigenetic alterations and corrects pulmonary pathology in congenital diaphragmatic hernia
|
Ullrich, Sarah J.
|
|
|
32 |
C |
p. 594-602
|
artikel
|
| 35 |
In vivo genome editing for hemophilia B therapy by the combination of rebalancing and therapeutic gene knockin using a viral and non-viral vector
|
Lee, Jeong Hyeon
|
|
|
32 |
C |
p. 161-172
|
artikel
|
| 36 |
IPs-GRUAtt: An attention-based bidirectional gated recurrent unit network for predicting phosphorylation sites of SARS-CoV-2 infection
|
Zhang, Guiyang
|
|
|
32 |
C |
p. 28-35
|
artikel
|
| 37 |
Landscape and perturbation of enhancer-driven core transcription regulatory circuits in cancer
|
Xu, Juan
|
|
|
32 |
C |
p. 872-874
|
artikel
|
| 38 |
Landscape and significance of human super enhancer-driven core transcription regulatory circuitry
|
Feng, Chenchen
|
|
|
32 |
C |
p. 385-401
|
artikel
|
| 39 |
Lateral flow immunoassay for rapid and sensitive detection of dsRNA contaminants in in vitro-transcribed mRNA products
|
Luo, Dengwang
|
|
|
32 |
C |
p. 445-453
|
artikel
|
| 40 |
Lentiviral vectors for inducible, transactivator-free advanced therapy medicinal products: Application to CAR-T cells
|
Tristán-Manzano, María
|
|
|
32 |
C |
p. 322-339
|
artikel
|
| 41 |
Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection
|
Byrnes, Amy E.
|
|
|
32 |
C |
p. 773-793
|
artikel
|
| 42 |
Lipid nanoparticle-encapsulated, chemically modified anti-adenoviral siRNAs inhibit hepatic adenovirus infection in immunosuppressed Syrian hamsters
|
Geisler, Anja
|
|
|
32 |
C |
p. 923-936
|
artikel
|
| 43 |
Liver lobe-specific hydrodynamic gene delivery to baboons: A preclinical trial for hemophilia gene therapy
|
Kamimura, Kenya
|
|
|
32 |
C |
p. 903-913
|
artikel
|
| 44 |
LncRNA ZNF593-AS alleviates diabetic cardiomyopathy via suppressing IRF3 signaling pathway
|
Xie, Rong
|
|
|
32 |
C |
p. 689-703
|
artikel
|
| 45 |
Mechanical stimuli-induced CCL2 restores adult mouse cells to regenerate hair follicles
|
Wu, Wang
|
|
|
32 |
C |
p. 94-110
|
artikel
|
| 46 |
Mechanisms of NAT10 as ac4C writer in diseases
|
Xie, Lihua
|
|
|
32 |
C |
p. 359-368
|
artikel
|
| 47 |
MicroRNA-nanoparticles against cancer: Opportunities and challenges for personalized medicine
|
Martino, Elisa
|
|
|
32 |
C |
p. 371-384
|
artikel
|
| 48 |
MiR34 contributes to spinal muscular atrophy and AAV9-mediated delivery of MiR34a ameliorates the motor deficits in SMA mice
|
Chen, Tai-Heng
|
|
|
32 |
C |
p. 144-160
|
artikel
|
| 49 |
miR-342-5p downstream to Notch enhances arterialization of endothelial cells in response to shear stress by repressing MYC
|
Zhang, Xiaoyan
|
|
|
32 |
C |
p. 343-358
|
artikel
|
| 50 |
Mitochondrial genome editing: Get aCcess to modeling broad disease mutations with engineered base editors
|
Xu, Chunlong
|
|
|
32 |
C |
p. 566-567
|
artikel
|
| 51 |
Modified ASO conjugates encapsulated with cytidinyl/cationic lipids exhibit more potent and longer-lasting anti-HCC effects
|
Pan, Yufei
|
|
|
32 |
C |
p. 807-821
|
artikel
|
| 52 |
MutSα and MutSβ as size-dependent cellular determinants for prime editing in human embryonic stem cells
|
Park, Ju-Chan
|
|
|
32 |
C |
p. 914-922
|
artikel
|
| 53 |
Novel Fas-TNFR chimeras that prevent Fas ligand-mediated kill and signal synergistically to enhance CAR T cell efficacy
|
McKenzie, Callum
|
|
|
32 |
C |
p. 603-621
|
artikel
|
| 54 |
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies
|
Brusson, Mégane
|
|
|
32 |
C |
p. 229-246
|
artikel
|
| 55 |
Orchestrating epigenetic readers: Progress in understanding the functions of bromodomain-containing protein 4 complexes
|
Brasier, Allan R.
|
|
|
32 |
C |
p. 340-342
|
artikel
|
| 56 |
Oversized liposomes boost macrophage-targeted RNA delivery to regulate macrophage polarity
|
Wang, Dongqing
|
|
|
32 |
C |
p. 877-878
|
artikel
|
| 57 |
Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice
|
Saoudi, Amel
|
|
|
32 |
C |
p. 173-188
|
artikel
|
| 58 |
Poly(β-amino ester)s-based nanovehicles: Structural regulation and gene delivery
|
Zhang, Jiayu
|
|
|
32 |
C |
p. 568-581
|
artikel
|
| 59 |
Positive feedback loop of miR-320 and CD36 regulates the hyperglycemic memory-induced diabetic diastolic cardiac dysfunction
|
Kankuri, Esko
|
|
|
32 |
C |
p. 318-321
|
artikel
|
| 60 |
PPFIA1-targeting miR-181a mimic and saRNA overcome imatinib resistance in BCR-ABL1-independent chronic myeloid leukemia by suppressing leukemia stem cell regeneration
|
Su, Rui
|
|
|
32 |
C |
p. 729-742
|
artikel
|
| 61 |
Proof-of-concept study for liver-directed miQURE technology in a dyslipidemic mouse model
|
Zancanella, Vanessa
|
|
|
32 |
C |
p. 454-467
|
artikel
|
| 62 |
Rationale and strategies for the development of safe and effective optimized AAV vectors for human gene therapy
|
Srivastava, Arun
|
|
|
32 |
C |
p. 949-959
|
artikel
|
| 63 |
Resistance to nirmatrelvir due to mutations in the Mpro in the subvariants of SARS-CoV-2 Omicron: Another concern?
|
Chatterjee, Srijan
|
|
|
32 |
C |
p. 263-266
|
artikel
|
| 64 |
Restoration of brain dystrophin using tricyclo-DNA ASOs restores neurobehavioral deficits in DMD mice
|
Karuppasamy, Muthukumar
|
|
|
32 |
C |
p. 841-842
|
artikel
|
| 65 |
Retraction Notice to: MicroRNA-494 Inhibits the LRG1 Expression to Induce Proliferation and Migration of VECs in Rats following Myocardial Infarction
|
Su, Qiang
|
|
|
32 |
C |
p. 667
|
artikel
|
| 66 |
Role of miR-300-3p in Leydig cell function and differentiation: A therapeutic target for obesity-related testosterone deficiency
|
Liang, Jinlian
|
|
|
32 |
C |
p. 879-895
|
artikel
|
| 67 |
Self-replicating RNA nanoparticle vaccine elicits protective immune responses against SARS-CoV-2
|
Lin, Guibin
|
|
|
32 |
C |
p. 650-666
|
artikel
|
| 68 |
Self-transfecting GMO-PMO chimera targeting Nanog enable gene silencing in vitro and suppresses tumor growth in 4T1 allografts in mouse
|
Das, Ujjal
|
|
|
32 |
C |
p. 203-228
|
artikel
|
| 69 |
Silencing of microRNA-106b-5p prevents doxorubicin-mediated cardiotoxicity through modulation of the PR55α/YY1/sST2 signaling axis
|
Lax, Antonio
|
|
|
32 |
C |
p. 704-720
|
artikel
|
| 70 |
SINEUP non-coding RNA activity depends on specific N6-methyladenosine nucleotides
|
Pierattini, Bianca
|
|
|
32 |
C |
p. 402-414
|
artikel
|
| 71 |
Single-swap editing for the correction of common Duchenne muscular dystrophy mutations
|
Chai, Andreas C.
|
|
|
32 |
C |
p. 522-535
|
artikel
|
| 72 |
siRNA targeting ANGPTL3 stands in the spotlight for lipid-lowering therapy
|
Hu, Bo
|
|
|
32 |
C |
p. 369-370
|
artikel
|
| 73 |
Specific DMPK-promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells
|
Porquet, Florent
|
|
|
32 |
C |
p. 857-871
|
artikel
|
| 74 |
STAT3 silencing by an aptamer-based strategy hampers the crosstalk between NSCLC cells and cancer-associated fibroblasts
|
Ibba, Maria L.
|
|
|
32 |
C |
p. 111-126
|
artikel
|
| 75 |
Strategic self-limiting production of infectious HIV particles by CRISPR in permissive cells
|
Liu, Hong
|
|
|
32 |
C |
p. 1010-1025
|
artikel
|
| 76 |
Strategies for precise gene edits in mammalian cells
|
Fichter, Katye M.
|
|
|
32 |
C |
p. 536-552
|
artikel
|
| 77 |
Streamlined DNA-encoded small molecule library screening and validation for the discovery of novel chemotypes targeting BET proteins
|
Jeong, Seoyeon
|
|
|
32 |
C |
p. 637-649
|
artikel
|
| 78 |
Synthetic miR-21 decoy circularized by tRNA splicing mechanism inhibited tumorigenesis in glioblastoma in vitro and in vivo models
|
Bayat, Hadi
|
|
|
32 |
C |
p. 432-444
|
artikel
|
| 79 |
Targeting microRNA-10 in glioma; a focus with potential therapeutic application in genome editing
|
Maus, Katharina
|
|
|
32 |
C |
p. 504-506
|
artikel
|
| 80 |
Terminal bridging of siRNA duplex at the ribose 2′ position controls strand bias and target sequence preference
|
Shibata, Atsushi
|
|
|
32 |
C |
p. 468-477
|
artikel
|
| 81 |
The impact of nucleoside base modification in mRNA vaccine is influenced by the chemistry of its lipid nanoparticle delivery system
|
Bernard, Marie-Clotilde
|
|
|
32 |
C |
p. 794-806
|
artikel
|
| 82 |
The miR-148/152 family contributes to angiogenesis of human pluripotent stem cell- derived endothelial cells by inhibiting MEOX2
|
Ding, Fengyue
|
|
|
32 |
C |
p. 582-593
|
artikel
|
| 83 |
Transient and tunable CRISPRa regulation of APOBEC/AID genes for targeting hepatitis B virus
|
Kostyushev, Dmitry
|
|
|
32 |
C |
p. 478-493
|
artikel
|
Tijdschrift beschrijving