| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAV-mediated GBA1 and GDNF rescue neurological defects in a murine model of neuronopathic Gaucher disease
|
Meng, Yuan
|
|
|
36 |
2 |
p.
|
artikel
|
| 2 |
AAVone: A cost-effective, single-plasmid solution for efficient AAV production with reduced DNA impurities
|
Yang, Rongze
|
|
|
36 |
2 |
p.
|
artikel
|
| 3 |
A CRISPR/Cas9 library screening identifies CARM1 as a critical inhibitor of ferroptosis in hepatocellular carcinoma cells
|
Cheng, Yiming
|
|
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36 |
2 |
p.
|
artikel
|
| 4 |
A4GALT-targeting siRNA lipid nanoparticles ameliorate Fabry disease phenotype: Greater efficacy in endothelial cells than in podocytes
|
Shin, Yoo Jin
|
|
|
36 |
2 |
p.
|
artikel
|
| 5 |
A Lyme disease mRNA vaccine targeting Borrelia burgdorferi OspA induces strong immune responses and prevents transmission in mice
|
Tahir, Djamel
|
|
|
36 |
2 |
p.
|
artikel
|
| 6 |
An inflammation-associated lncRNA induces neuronal damage via mitochondrial dysfunction
|
Olazagoitia-Garmendia, Ane
|
|
|
36 |
2 |
p.
|
artikel
|
| 7 |
A novel miniaturized filamentous phagemid as a gene delivery vehicle to target mammalian cells
|
Wong, Shirley
|
|
|
36 |
2 |
p.
|
artikel
|
| 8 |
An oversized AAV8 vector to deliver CPS1
|
Li, Shuang
|
|
|
36 |
2 |
p.
|
artikel
|
| 9 |
Antibody and aptamer-based therapies for osteoarthritis: Application of antibodies and promise of aptamers
|
Chen, Huaizhi
|
|
|
36 |
2 |
p.
|
artikel
|
| 10 |
Anti-gene oligonucleotides targeting Friedreich’s ataxia expanded GAA⋅TTC repeats increase Frataxin expression
|
Mozafari, Negin
|
|
|
36 |
2 |
p.
|
artikel
|
| 11 |
Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects
|
Hara, Tomoaki
|
|
|
36 |
2 |
p.
|
artikel
|
| 12 |
A single stereogenic center matters: Development of stereodefined anti-tau PMO-gapmers
|
Chen, Suxiang
|
|
|
36 |
2 |
p.
|
artikel
|
| 13 |
Bile acid accumulation induced by miR-122 deficiency in liver parenchyma promotes cancer cell growth in hepatocellular carcinoma
|
Huang, Jia-Hui
|
|
|
36 |
2 |
p.
|
artikel
|
| 14 |
Binding of G-quadruplex DNA and serum albumins by synthetic non-proteinogenic amino acids: Implications for c-Myc-related anticancer activity and drug delivery
|
Simonyan, Hayarpi
|
|
|
36 |
2 |
p.
|
artikel
|
| 15 |
CAG-targeted brain-permeable therapy tested in biallelic humanized polyQ mouse models
|
Surdyka, Magdalena
|
|
|
36 |
2 |
p.
|
artikel
|
| 16 |
CircITSN1/EIF4A3/Itsn1 axis mediates postoperative cognitive dysfunction in aged mice: A novel mechanism and therapeutic target
|
Zhang, Changteng
|
|
|
36 |
2 |
p.
|
artikel
|
| 17 |
CLCluster: A redundancy-reduction contrastive learning-based clustering method of cancer subtype based on multi-omics data
|
Wang, Hong
|
|
|
36 |
2 |
p.
|
artikel
|
| 18 |
Closing the gap: Nonviral TFAMoplex transfection boosted by bZIP domains compared to AAV-mediated transduction
|
Honrath, Steffen
|
|
|
36 |
2 |
p.
|
artikel
|
| 19 |
Conjugated STING agonists
|
Qu, Shuhao
|
|
|
36 |
2 |
p.
|
artikel
|
| 20 |
Correcting tau isoform ratios with a long-acting antisense oligonucleotide alleviates 4R-tauopathy phenotypes
|
Iwata-Endo, Kuniyuki
|
|
|
36 |
2 |
p.
|
artikel
|
| 21 |
CRISPR antiviral inhibits neurotrophic JC polyomavirus in 2D and 3D culture models through dual-gRNA excision by SaCas9
|
Rocchi, Angela
|
|
|
36 |
2 |
p.
|
artikel
|
| 22 |
CRISPR-directed exon skipping; friend or foe (opportunity or warning sign)?
|
Banas, Kelly H.
|
|
|
36 |
2 |
p.
|
artikel
|
| 23 |
CyKILR splice variants: A double-edged sword in non-small cell lung cancer
|
Khan, Muhammad Riaz
|
|
|
36 |
2 |
p.
|
artikel
|
| 24 |
Deciphering the NSP12/7/8 complex: Key insights into coronavirus replication and potential therapeutic targets
|
Luwen, Gao
|
|
|
36 |
2 |
p.
|
artikel
|
| 25 |
Deconvolution of cargo delivery and immunogenicity following intranasal delivery of mRNA lipid nanoparticle vaccines
|
Vu, Mai N.
|
|
|
36 |
2 |
p.
|
artikel
|
| 26 |
Design and screening of novel endosomal escape compounds that enhance functional delivery of oligonucleotides in vitro
|
Estupiñán, H. Yesid
|
|
|
36 |
2 |
p.
|
artikel
|
| 27 |
Design, pharmacology, and toxicology of a novel chemically modified siRNA targeting hepatic angiotensinogen
|
Huang, Ze-Ao
|
|
|
36 |
2 |
p.
|
artikel
|
| 28 |
Differentially expressed ncRNAs as key regulators in infection of human bronchial epithelial cells by the SARS-CoV-2 Delta variant
|
Ranches, Glory
|
|
|
36 |
2 |
p.
|
artikel
|
| 29 |
Durable HTT silencing using non-evolved dCas9 epigenome editors in patient-derived cells
|
Waldo, Jennifer J.
|
|
|
36 |
2 |
p.
|
artikel
|
| 30 |
Editing out the polymers: Toward a gene therapy for FENIB
|
Miranda, Elena
|
|
|
36 |
2 |
p.
|
artikel
|
| 31 |
Enhancing mRNA translation efficiency by introducing sequence optimized AU-rich elements in 3′ UTR via HuR anchorage
|
Ma, Xinghuan
|
|
|
36 |
2 |
p.
|
artikel
|
| 32 |
Fighting RNA viruses with a gold nanoparticle Cas13d gene-editing armor
|
De Carli, Alessandro
|
|
|
36 |
2 |
p.
|
artikel
|
| 33 |
FUBP3: A new player in HIV-1 transcriptional activation and immune regulation
|
Marichannegowda, Manukumar Honnayakanahalli
|
|
|
36 |
2 |
p.
|
artikel
|
| 34 |
FUBP3 enhances HIV-1 transcriptional activity and regulates immune response pathways in T cells
|
Gibaut, Quentin M.R.
|
|
|
36 |
2 |
p.
|
artikel
|
| 35 |
FUBP3 enhances HIV-1 transcriptional activity and regulates immune response pathways in T cells
|
Gibaut, Quentin M.R.
|
|
|
36 |
2 |
p.
|
artikel
|
| 36 |
Generative artificial intelligence, integrative bioinformatics, and single-cell analysis reveal Alzheimer’s genetic and immune landscape
|
Das, Arpita
|
|
|
36 |
2 |
p.
|
artikel
|
| 37 |
Genetic adjuvants: A paradigm shift in vaccine development and immune modulation
|
Spunde, Karina
|
|
|
36 |
2 |
p.
|
artikel
|
| 38 |
Harnessing endogenous miRNA targeting ZIKV: A cutting-edge strategy to inhibit virus infection
|
Rosa, Rhubia S.M.
|
|
|
36 |
2 |
p.
|
artikel
|
| 39 |
Harnessing nc886 to overcome immune and apoptotic barriers in adenoviral gene therapy
|
Hammad, Mohamed
|
|
|
36 |
2 |
p.
|
artikel
|
| 40 |
High-throughput 3D spheroid screens identify microRNA sensitizers for improved thermoradiotherapy in locally advanced cancers
|
Xu, MengFei
|
|
|
36 |
2 |
p.
|
artikel
|
| 41 |
Hyperandrogen-induced imbalance of FOXO4-AR regulatory loop contributes to ovulatory disorders in polycystic ovary syndrome
|
Zhou, Ruiqiong
|
|
|
36 |
2 |
p.
|
artikel
|
| 42 |
Inhibition of IFNAR-JAK signaling enhances tolerability and transgene expression of systemic non-viral DNA delivery
|
Senapati, Sujata
|
|
|
36 |
2 |
p.
|
artikel
|
| 43 |
Inhibition of the LINE1-derived MET transcript induces apoptosis and oncoprotein knockdown in cancer cells
|
Miglio, Umberto
|
|
|
36 |
2 |
p.
|
artikel
|
| 44 |
In vivo base editing of Angptl3 via lipid nanoparticles to treat cardiovascular disease
|
Khirallah, Jennifer
|
|
|
36 |
2 |
p.
|
artikel
|
| 45 |
Leveraging cardiac reprogramming with CRISPRa-SAM technology
|
Borthakur, Atreyee
|
|
|
36 |
2 |
p.
|
artikel
|
| 46 |
Ligand-modified rAAV6 vectors with nanoblades allow high-level gene knockin in HSPCs without compromising cell survival
|
Gutierrez-Guerrero, Alejandra
|
|
|
36 |
2 |
p.
|
artikel
|
| 47 |
Looking beyond the origin of SARS-CoV-2: Significant strategic aspects during the five-year journey of COVID-19 vaccine development
|
Chakraborty, Chiranjib
|
|
|
36 |
2 |
p.
|
artikel
|
| 48 |
Mechanistic intracellular PK/PD modeling to inform development strategies for small interfering RNA therapeutics
|
Chen, Lin
|
|
|
36 |
2 |
p.
|
artikel
|
| 49 |
microRNA-133a as an indicator of disease progression and treatment response in X-linked myotubular myopathy
|
Maani, Nika
|
|
|
36 |
2 |
p.
|
artikel
|
| 50 |
MiR-34 at the crossroads of SMA pathogenesis and therapy: Emerging biomarker and therapeutic target
|
Chen, Jun-An
|
|
|
36 |
2 |
p.
|
artikel
|
| 51 |
miRNA-642a-3p protects β cells from glucolipotoxicity
|
Pinhanços, Sandra Sofia
|
|
|
36 |
2 |
p.
|
artikel
|
| 52 |
mRNA-encoded ACE2 decoy lipid nanoparticles for neutralizing SARS-CoV-2 variants
|
Guimarães, Lays Cordeiro
|
|
|
36 |
2 |
p.
|
artikel
|
| 53 |
Nanoparticle-formulated mRNA encoding engineered multivalent SIRPα-Fc fusion proteins shows robust anti-cancer activity in preclinical models
|
Lal, Shruti
|
|
|
36 |
2 |
p.
|
artikel
|
| 54 |
Novel mutation SLFN14 T853fs associated with inherited macrothrombocytopenia
|
Xie, Haixiao
|
|
|
36 |
2 |
p.
|
artikel
|
| 55 |
Novel PEI-aldehyde conjugates for gene delivery: Promoting chondrogenic differentiation in human mesenchymal stem cells
|
Miranda-Balbuena, Diego
|
|
|
36 |
2 |
p.
|
artikel
|
| 56 |
Optimization of mtDNA-targeted platinum TALENs for bi-directionally modifying heteroplasmy levels in patient-derived m.3243A>G-iPSCs
|
Yahata, Naoki
|
|
|
36 |
2 |
p.
|
artikel
|
| 57 |
Optimized genomic editing of a common Duchenne muscular dystrophy mutation in patient-derived muscle cells and a new humanized mouse model
|
Durbacz, Mateusz Z.
|
|
|
36 |
2 |
p.
|
artikel
|
| 58 |
PAI1 regulating CHRNA1 contributes to primary focal hyperhidrosis: Clinical and experimental studies
|
Zheng, Ru-Jie
|
|
|
36 |
2 |
p.
|
artikel
|
| 59 |
Papillomavirus-like particles as vectors for ex vivo gene therapy of the skin
|
Diversi, Francesco
|
|
|
36 |
2 |
p.
|
artikel
|
| 60 |
Physicochemical and functional assessment of messenger RNA 5′Cap-end impurities under forced degradation conditions
|
Hutchinson, J.
|
|
|
36 |
2 |
p.
|
artikel
|
| 61 |
Removal of dsRNA byproducts using affinity chromatography
|
Clark, Nathaniel E.
|
|
|
36 |
2 |
p.
|
artikel
|
| 62 |
Retraction Notice to: LncRNA LINC00963 Promotes Tumorigenesis and Radioresistance in Breast Cancer by Sponging miR-324-3p and Inducing ACK1 Expression
|
Zhang, Na
|
|
|
36 |
2 |
p.
|
artikel
|
| 63 |
Retraction Notice to: uc.454 Inhibited Growth by Targeting Heat Shock Protein Family A Member 12B in Non-Small-Cell Lung Cancer
|
Zhou, Jun
|
|
|
36 |
2 |
p.
|
artikel
|
| 64 |
Reverse-phase chromatography removes double-stranded RNA, fragments, and residual template to decrease immunogenicity and increase cell potency of mRNA and saRNA
|
Krušič, Andreja
|
|
|
36 |
2 |
p.
|
artikel
|
| 65 |
RNAa: Mechanisms, therapeutic potential, and clinical progress
|
Qian, Yukang
|
|
|
36 |
2 |
p.
|
artikel
|
| 66 |
RNA modifications at the heart of oral inflammation
|
Guzzi, Nicola
|
|
|
36 |
2 |
p.
|
artikel
|
| 67 |
SHMT2 is essential for mammalian preimplantation embryonic development through de novo biosynthesis of nucleotide metabolites
|
Shi, Mingze
|
|
|
36 |
2 |
p.
|
artikel
|
| 68 |
Single-guide RNA Cas9 and enhanced-deletion Cas9 rescue a recurrent USH2A-related splicing defect
|
De Angeli, Pietro
|
|
|
36 |
2 |
p.
|
artikel
|
| 69 |
Single-stranded HDR templates with truncated Cas12a-binding sequences improve knock-in efficiencies in primary human T cells
|
Nitulescu, Ana-Maria
|
|
|
36 |
2 |
p.
|
artikel
|
| 70 |
Splicing correction by peptide-conjugated morpholinos as a novel treatment for late-onset Pompe disease
|
Oliver, Ryan A.
|
|
|
36 |
2 |
p.
|
artikel
|
| 71 |
SPRINGing forward: Advancing RNA editing efficiency and precision with engineered ADAR2
|
Melzer, Nico
|
|
|
36 |
2 |
p.
|
artikel
|
| 72 |
Targeted mRNA delivery with bispecific antibodies that tether LNPs to cell surface markers
|
Dietmair, Bettina
|
|
|
36 |
2 |
p.
|
artikel
|
| 73 |
Targeting CIDEB alleviates liver steatosis and fibrosis in mouse MASH models
|
Lin, Yingying
|
|
|
36 |
2 |
p.
|
artikel
|
| 74 |
Targeting FANCM by antisense oligonucleotides in ALT-positive cancers
|
Tieo, Galen
|
|
|
36 |
2 |
p.
|
artikel
|
| 75 |
Targeting FANCM using the antisense oligonucleotides to treat the ALT-positive cancers
|
Naji, Shakir
|
|
|
36 |
2 |
p.
|
artikel
|
| 76 |
The interactions of Pu22 G-quadruplex, derived from c-MYC promoter sequence, with antitumor acridine derivatives—An NMR/MD combined study
|
Laskowski, Tomasz
|
|
|
36 |
2 |
p.
|
artikel
|
| 77 |
Validation of tRNA-derived fragments as diagnostic biomarkers in suspected acute stroke; limitations in analysis and quantification methods
|
Woudenberg, Tamar
|
|
|
36 |
2 |
p.
|
artikel
|
| 78 |
We ARE boosting translation: AU-rich elements for enhanced therapeutic mRNA translation
|
Froechlich, Guendalina
|
|
|
36 |
2 |
p.
|
artikel
|
| 79 |
Whole-transcriptome sequencing in neural and non-neural tissues of a mouse model identifies miR-34a as a key regulator in SMA pathogenesis
|
Wu, Liucheng
|
|
|
36 |
2 |
p.
|
artikel
|
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