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Cardiac vein retroinjections provide an efficient approach for global left ventricular gene transfer with adenovirus and adeno-associated virus
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QR-1011 restores defective ABCA4 splicing caused by multiple severe ABCA4 variants underlying Stargardt disease
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Expression of a large coding sequence: Gene therapy vectors for Ataxia Telangiectasia
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Elucidation of lipid nanoparticle surface structure in mRNA vaccines
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Design of hypoxia responsive CRISPR-Cas9 for target gene regulation
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Characteristics of FXa-storing platelets in hemophilia B mice and the influence of alcohol on the platelets
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High spontaneous integration rates of end-modified linear DNAs upon mammalian cell transfection
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Upregulation of alveolar fluid clearance is not sufficient for Na+,K+-ATPase β subunit-mediated gene therapy of LPS-induced acute lung injury in mice
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Full-length human dystrophin on human artificial chromosome compensates for mouse dystrophin deficiency in a Duchenne muscular dystrophy mouse model
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CRISPR/Cas9 mediated specific ablation of vegfa in retinal pigment epithelium efficiently regresses choroidal neovascularization
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Machine learning and metabolic modelling assisted implementation of a novel process analytical technology in cell and gene therapy manufacturing
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CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe disease
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Effects of pre-existing anti-adenovirus antibodies on transgene expression levels and therapeutic efficacies of arming oncolytic adenovirus
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Artificial intelligence workflow quantifying muscle features on Hematoxylin–Eosin stained sections reveals dystrophic phenotype amelioration upon treatment
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Overexpression of VEGF in dermal fibroblast cells accelerates the angiogenesis and wound healing function: in vitro and in vivo studies
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Genetic correction of haemoglobin E in an immortalised haemoglobin E/beta-thalassaemia cell line using the CRISPR/Cas9 system
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Delivery and assessment of a CRISPR/nCas9-based genome editing system on in vitro models of mucopolysaccharidoses IVA assisted by magnetite-based nanoparticles
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p63 silencing induces epigenetic modulation to enhance human cardiac fibroblast to cardiomyocyte-like differentiation
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Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription
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HideRNAs protect against CRISPR-Cas9 re-cutting after successful single base-pair gene editing
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Stent-based delivery of AAV2 vectors encoding oxidation-resistant apoA1
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Transiently expressed CRISPR/Cas9 induces wild-type dystrophin in vitro in DMD patient myoblasts carrying duplications
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Tumor RNA transfected DCs derived from iPS cells elicit cytotoxicity against cancer cells induced from colorectal cancer patients in vitro
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Therapeutic potential of highly functional codon-optimized microutrophin for muscle-specific expression
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RGEN-seq for highly sensitive amplification-free screen of off-target sites of gene editors
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Blood phenylalanine reduction reverses gene expression changes observed in a mouse model of phenylketonuria
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Functional roles of the membrane-associated AAV protein MAAP
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A combined treatment regimen of MGMT-modified γδ T cells and temozolomide chemotherapy is effective against primary high grade gliomas
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Gene transfer of MRCKα rescues lipopolysaccharide-induced acute lung injury by restoring alveolar capillary barrier function
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A guide in lentiviral vector production for hard-to-transfect cells, using cardiac-derived c-kit expressing cells as a model system
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Mesenchymal stromal cells as carriers of IL-12 reduce primary and metastatic tumors of murine melanoma
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Lentiviral and AAV-mediated expression of palivizumab offer protection against Respiratory Syncytial Virus infection
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Viral-mediated transduction of auditory neurons with opsins for optical and hybrid activation
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Comprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering
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Epigenetic silencing directs expression heterogeneity of stably integrated multi-transcript unit genetic circuits
- Jan Zimak
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Electroporation outperforms in vivo-jetPEI for intratumoral DNA-based reporter gene transfer
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High-throughput continuous-flow microfluidic electroporation of mRNA into primary human T cells for applications in cellular therapy manufacturing
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Genome targeting by hybrid Flp-TAL recombinases
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RNA editing of BFP, a point mutant of GFP, using artificial APOBEC1 deaminase to restore the genetic code
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Viromers as carriers for mRNA-mediated expression of therapeutic molecules under inflammatory conditions
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Rapid repair of human disease-specific single-nucleotide variants by One-SHOT genome editing
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DNA unchained: two assays to discover and study inhibitors of the DNA clustering function of barrier-to-autointegration factor
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Transcriptional repression of PTEN in neural cells using CRISPR/dCas9 epigenetic editing
- C. Moses
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Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34+ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin
- Andrés Lamsfus-Calle
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Adeno-associated virus-binding antibodies detected in cats living in the Northeastern United States lack neutralizing activity
- Kei Adachi
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Density functional theory studies on cytosine analogues for inducing double-proton transfer with guanine
- Jinjie Xue
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TALEN mediated gene editing in a mouse model of Fanconi anemia
- Maria José Pino-Barrio
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Specificity profiling of CRISPR system reveals greatly enhanced off-target gene editing
- Yao Wang
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Investigation and Restoration of BEST1 Activity in Patient-derived RPEs with Dominant Mutations
- Changyi Ji
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Gene targeting in adult organs using in vivo cleavable donor plasmids for CRISPR-Cas9 and CRISPR-Cas12a