| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAV-regulated Serpine2 overexpression promotes hair cell regeneration
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Sun, Qiuhan
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35 |
4 |
p.
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artikel
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| 2 |
A drug candidate for a rare polyalanine disease targeting the protein quality control
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Pochtar, Sharon
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35 |
4 |
p.
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artikel
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| 3 |
A dual functional mucus- and cell-penetrating peptide enhances mRNA lipid nanoparticle delivery to the lung
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Kumar, Sahana
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35 |
4 |
p.
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artikel
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| 4 |
A lipid nanoparticle-based oligodendrocyte-specific mRNA therapy
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Sawamura, Masanori
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35 |
4 |
p.
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artikel
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| 5 |
Allele-specific CRISPR-Cas9 editing inactivates a single-nucleotide variant associated with collagen VI muscular dystrophy
|
Aoki, Yoshitsugu
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35 |
4 |
p.
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artikel
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| 6 |
Alternative splicing dysregulation across tissue and therapeutic approaches in a mouse model of myotonic dystrophy type 1
|
Hicks, Sawyer M.
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35 |
4 |
p.
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artikel
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| 7 |
A modular platform for nucleic acid-driven multimerization of nanobodies for advanced molecular imaging
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Rebolledo, Laura P.
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35 |
4 |
p.
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artikel
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| 8 |
A novel hyperactive variant of the Sleeping Beauty transposase facilitates non-viral genome engineering
|
Ochmann, Matthias Thomas
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35 |
4 |
p.
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artikel
|
| 9 |
Anti-gene oligonucleotide clamps invade dsDNA and downregulate huntingtin expression
|
Umek, Tea
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35 |
4 |
p.
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artikel
|
| 10 |
AptamerRunner: An accessible aptamer structure prediction and clustering algorithm for visualization of selected aptamers
|
Ruiz-Ciancio, Dario
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35 |
4 |
p.
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artikel
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| 11 |
A route to engineer a genome editor for gene therapy
|
Yu, Wenxia
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35 |
4 |
p.
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artikel
|
| 12 |
Boosting CRISPR-Cas9 efficiency through enhanced homologous recombination
|
Kim, So Hyun
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35 |
4 |
p.
|
artikel
|
| 13 |
Breaking barriers: Aptamers in ocular disease treatment
|
Wang, Tao
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35 |
4 |
p.
|
artikel
|
| 14 |
Cas9 editing of ATXN1 in a spinocerebellar ataxia type 1 mice and human iPSC-derived neurons
|
Fagan, Kelly J.
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35 |
4 |
p.
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artikel
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| 15 |
Cell-specific delivery of CRISPR-Cas9 with pseudotyped lentiviral particles: Just change the envelope
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Covo-Vergara, Ángela
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35 |
4 |
p.
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artikel
|
| 16 |
Cell-targeted gene modification by delivery of CRISPR-Cas9 ribonucleoprotein complexes in pseudotyped lentivirus-derived nanoparticles
|
Nielsen, Ian Helstrup
|
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35 |
4 |
p.
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artikel
|
| 17 |
CHD6 eviction of promoter nucleosomes maintains housekeeping transcriptional program in prostate cancer
|
Bu, Lina
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35 |
4 |
p.
|
artikel
|
| 18 |
CRISPR-Cas9-directed gene therapy for spinocerebellar ataxia type 1
|
Miclăuș, Mihai
|
|
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35 |
4 |
p.
|
artikel
|
| 19 |
CRISPR-Cas13d: RNA’s own Jedi Master in the fight against viral darkness
|
Kontarakis, Zacharias
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35 |
4 |
p.
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artikel
|
| 20 |
CRISPR-Cas9-mediated homology-directed repair for precise gene editing
|
Liao, Hongyu
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35 |
4 |
p.
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artikel
|
| 21 |
Delivering therapeutic RNA into the brain using extracellular vesicles
|
Shirmast, Paniz
|
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35 |
4 |
p.
|
artikel
|
| 22 |
Direct cardiac reprogramming via combined CRISPRa-mediated endogenous Gata4 activation and exogenous Mef2c and Tbx5 expression
|
Huang, Peisen
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35 |
4 |
p.
|
artikel
|
| 23 |
Direct delivery of Cas9 or base editor protein and guide RNA complex enables genome editing in the retina
|
Pulman, Juliette
|
|
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35 |
4 |
p.
|
artikel
|
| 24 |
Discovery of peptides for ligand-mediated delivery of mRNA lipid nanoparticles to cystic fibrosis lung epithelia
|
Soto, Melissa R.
|
|
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35 |
4 |
p.
|
artikel
|
| 25 |
DNA aptamers that modulate biological activity of model neurons
|
Rolli, Jenelle
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35 |
4 |
p.
|
artikel
|
| 26 |
DNA-PKcs inhibition improves sequential gene insertion of the full-length CFTR cDNA in airway stem cells
|
Stack, Jacob T.
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35 |
4 |
p.
|
artikel
|
| 27 |
Double-stranded DNA invasion by anti-gene oligonucleotide clamps
|
De, Angana
|
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35 |
4 |
p.
|
artikel
|
| 28 |
DTMP-prime: A deep transformer-based model for predicting prime editing efficiency and PegRNA activity
|
Alipanahi, Roghayyeh
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|
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35 |
4 |
p.
|
artikel
|
| 29 |
Editing of homologous globin genes by nickase-deficient base editor mitigates large intergenic deletions in HSPCs
|
George, Anila
|
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35 |
4 |
p.
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artikel
|
| 30 |
Efficient and in situ correction of hemoglobin Constant Spring mutation by prime editing in human hematopoietic cells
|
Shao, Congwen
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|
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35 |
4 |
p.
|
artikel
|
| 31 |
Engineered AAV capsid transport mutants overcome transduction deficiencies in the aged CNS
|
Sandoval, Ivette M.
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35 |
4 |
p.
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artikel
|
| 32 |
Engineered packaging cell line for the enhanced production of baboon-enveloped retroviral vectors
|
Klatt, Denise
|
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35 |
4 |
p.
|
artikel
|
| 33 |
Exploring non-coding variants and evaluation of antisense oligonucleotides for splicing redirection in Usher syndrome
|
García-Bohórquez, Belén
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|
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35 |
4 |
p.
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artikel
|
| 34 |
Exploring the therapeutic potential of sγPNA-141: Pharmacodynamics and mechanistic insights during ischemic stroke recovery
|
Yadav, Sanjeev Kumar
|
|
|
35 |
4 |
p.
|
artikel
|
| 35 |
First-in-class transactivator-free, doxycycline-inducible IL-18-engineered CAR-T cells for relapsed/refractory B cell lymphomas
|
Justicia-Lirio, Pedro
|
|
|
35 |
4 |
p.
|
artikel
|
| 36 |
From classic to innovative: The continuation and modern application of calcium phosphate co-precipitation method in mRNA tumor delivery
|
Zhang, Jiayu
|
|
|
35 |
4 |
p.
|
artikel
|
| 37 |
HBx induces chemoresistance in diffuse large B cell lymphoma by inhibiting intrinsic apoptosis via the NF-κB/XIAP pathway
|
Zhan, Zhumei
|
|
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35 |
4 |
p.
|
artikel
|
| 38 |
Highly efficient nucleic acid encapsulation method for targeted gene therapy using antibody conjugation system
|
Hong, Seokbong
|
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35 |
4 |
p.
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artikel
|
| 39 |
Identification of a histone deacetylase inhibitor as a therapeutic candidate for congenital central hypoventilation syndrome
|
Africano, Chiara
|
|
|
35 |
4 |
p.
|
artikel
|
| 40 |
Inhaled aptamer therapy: A hopeful therapy for lung disease
|
Martin, M. Elena
|
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|
35 |
4 |
p.
|
artikel
|
| 41 |
Inhibition of SARS-CoV-2 growth in the lungs of mice by a peptide-conjugated morpholino oligomer targeting viral RNA
|
Sakai, Alexandra
|
|
|
35 |
4 |
p.
|
artikel
|
| 42 |
Integrating IL-12 mRNA nanotechnology with SBRT eliminates T cell exhaustion in preclinical models of pancreatic cancer
|
Hughson, Angela L.
|
|
|
35 |
4 |
p.
|
artikel
|
| 43 |
Interpretable deep cross networks unveiled common signatures of dysregulated epitranscriptomes across 12 cancer types
|
Xia, Rong
|
|
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35 |
4 |
p.
|
artikel
|
| 44 |
Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4
|
Vázquez-Domínguez, Irene
|
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|
35 |
4 |
p.
|
artikel
|
| 45 |
Local CpG-Stat3 siRNA treatment improves antitumor effects of immune checkpoint inhibitors
|
Zhang, Chunyan
|
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|
35 |
4 |
p.
|
artikel
|
| 46 |
l-Type amino acid transporter 1-targeting nanoparticles for antisense oligonucleotide delivery to the CNS
|
Lim, Yu Na
|
|
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35 |
4 |
p.
|
artikel
|
| 47 |
Mechanistic insights into ASO-RNA complexation: Advancing antisense oligonucleotide design strategies
|
Hörberg, Johanna
|
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35 |
4 |
p.
|
artikel
|
| 48 |
Messing up with the regulator: A rational AI-driven drug design strategy to disrupt miRNA-target interactions
|
Leitão, Ana Lúcia
|
|
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35 |
4 |
p.
|
artikel
|
| 49 |
Microcell-mediated chromosome transfer between non-identical human iPSCs
|
Uno, Narumi
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35 |
4 |
p.
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artikel
|
| 50 |
miR-125b Upregulates miR-34a and Sequentially Activates Stress Adaption and Cell Death Mechanisms in Multiple Myeloma
|
Misso, Gabriella
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35 |
4 |
p.
|
artikel
|
| 51 |
miRNA-199b-5p suppresses of oral squamous cell carcinoma by targeting apical-basolateral polarity via Scribble/Lgl
|
Kim, Shihyun
|
|
|
35 |
4 |
p.
|
artikel
|
| 52 |
Molecular classification of geriatric breast cancer displays distinct senescent subgroups of prognostic significance
|
Wu, Xia
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|
35 |
4 |
p.
|
artikel
|
| 53 |
mRNA vaccine-induced IgG mediates nasal SARS-CoV-2 clearance in mice
|
Fricke, Charlie
|
|
|
35 |
4 |
p.
|
artikel
|
| 54 |
Multi-gene duplication removal in an engineered human cellular MECP2 duplication syndrome model with an IRAK1-MECP2 duplication
|
Rizvi, Samar Z.
|
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35 |
4 |
p.
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artikel
|
| 55 |
Non-coding sabotage: How Gadlor lncRNAs hijack heart function
|
Hunkler, Hannah Jill
|
|
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35 |
4 |
p.
|
artikel
|
| 56 |
Novel readthrough agent suppresses nonsense mutations and restores functional type VII collagen and laminin 332 in epidermolysis bullosa
|
Levian, Brandon
|
|
|
35 |
4 |
p.
|
artikel
|
| 57 |
Optimization of a lentivirus-mediated gene therapy targeting HIV-1 RNA to eliminate HIV-1-infected cells
|
Buckingham, Amanda B.
|
|
|
35 |
4 |
p.
|
artikel
|
| 58 |
PAK1 Promotes the Proliferation and Inhibits Apoptosis of Human Spermatogonial Stem Cells via PDK1/KDR/ZNF367 and ERK1/2 and AKT Pathways
|
Fu, Hongyong
|
|
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35 |
4 |
p.
|
artikel
|
| 59 |
Precision and efficacy of RNA-guided DNA integration in high-expressing muscle loci
|
Padmaswari, Made Harumi
|
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|
35 |
4 |
p.
|
artikel
|
| 60 |
Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation
|
Miller, Rachael
|
|
|
35 |
4 |
p.
|
artikel
|
| 61 |
Profiling patent compounds in lipid nanoparticle formulations of siRNA
|
Han, Yunfeng
|
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35 |
4 |
p.
|
artikel
|
| 62 |
Quality by design for mRNA platform purification based on continuous oligo-dT chromatography
|
Qu, Jixin
|
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|
35 |
4 |
p.
|
artikel
|
| 63 |
RAB18 regulates extrahepatic siRNA-mediated gene silencing efficacy
|
Lu, Jiamiao
|
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35 |
4 |
p.
|
artikel
|
| 64 |
Rationalizing the effects of RNA modifications on protein interactions
|
Vandelli, Andrea
|
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35 |
4 |
p.
|
artikel
|
| 65 |
Rationally designed Campylobacter jejuni Cas9 enables efficient gene activation and base editing
|
Chen, Yuxi
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35 |
4 |
p.
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artikel
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| 66 |
Reading through the nonsense: Gentamicin and ELX-02 as rescuing therapies for epidermolysis bullosa
|
Anderson-Crannage, Morgan
|
|
|
35 |
4 |
p.
|
artikel
|
| 67 |
Rescue of the endogenous FVIII expression in hemophilia A mice using CRISPR-Cas9 mRNA LNPs
|
Chen, Chun-Yu
|
|
|
35 |
4 |
p.
|
artikel
|
| 68 |
Retraction Notice to: LncRNA PVT1 promotes tumorigenesis of glioblastoma by recruiting COPS5 to deubiquitinate and stabilize TRIM24
|
Lv, Tao
|
|
|
35 |
4 |
p.
|
artikel
|
| 69 |
Retraction Notice to: Overexpression of circRNA circUCK2 Attenuates Cell Apoptosis in Cerebral Ischemia-Reperfusion Injury via miR-125b-5p/GDF11 Signaling
|
Chen, Wanghao
|
|
|
35 |
4 |
p.
|
artikel
|
| 70 |
Ruvkun and Ambros recognized for miRNAs
|
Kay, Mark A.
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35 |
4 |
p.
|
artikel
|
| 71 |
Simple improvements in vector design afford substantial gains in AAV delivery of aggregation-slowing Aβ variants
|
Borgenheimer, Ella
|
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35 |
4 |
p.
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artikel
|
| 72 |
Targeting heterozygous dominant negative variant of KCNA2 using Gapmer ASO for the treatment of drug-resistant epilepsy
|
Huang, Hua
|
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35 |
4 |
p.
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artikel
|
| 73 |
Targeting ocular tissues with intravenously administered aptamers selected by in vivo SELEX
|
Korhonen, Sonja
|
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35 |
4 |
p.
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artikel
|
| 74 |
Targeting ZC3H11A elicits immunogenic cancer cell death through augmentation of antigen presentation and interferon response
|
Ali, Arwa
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35 |
4 |
p.
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artikel
|
| 75 |
Thank you to our 2024 reviewers
|
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35 |
4 |
p.
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artikel
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| 76 |
The beauty of symmetry: siRNA phosphorodithioate modifications reduce stereocomplexity, ease analysis, and can improve in vivo potency
|
Schöllkopf, Sophie
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35 |
4 |
p.
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artikel
|
| 77 |
The role of mRNA-galsomes and LNPs in enhancing HIV-specific T cell responses across various lymphoid organs
|
D’haese, Sigrid
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35 |
4 |
p.
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artikel
|
| 78 |
The role of RNA modifications in disease-associated macrophages
|
Huart, Camille
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35 |
4 |
p.
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artikel
|
| 79 |
The role of Sertoli cell-derived miR-143-3p in male fertility declines with age
|
Liang, Jinlian
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35 |
4 |
p.
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artikel
|
| 80 |
Triptolide Restores Autophagy to Alleviate Diabetic Renal Fibrosis through the miR-141-3p/PTEN/Akt/mTOR Pathway
|
Li, Xiao-yu
|
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35 |
4 |
p.
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artikel
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