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| Open AccessGene targeting in adult organs using in vivo cleavable donor plasmids for CRISPR-Cas9 and CRISPR-Cas12a
- Riki Ishibashi
- , Ritsuko Maki
- & Fumiko Toyoshima
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| Open AccessCardiac vein retroinjections provide an efficient approach for global left ventricular gene transfer with adenovirus and adeno-associated virus
- Jaakko Lampela
- , Juho Pajula
- & Seppo Ylä-Herttuala
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| Open AccessQR-1011 restores defective ABCA4 splicing caused by multiple severe ABCA4 variants underlying Stargardt disease
- Melita Kaltak
- , Petra de Bruijn
- & Jim Swildens
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| Open AccessExpression of a large coding sequence: Gene therapy vectors for Ataxia Telangiectasia
- Tanja Hirch
- , Nadine Brander
- & Ute Modlich
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| Open AccessElucidation of lipid nanoparticle surface structure in mRNA vaccines
- Mingzhang Maple Wang
- , Caitlin N. Wappelhorst
- & Qin Zou
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| Open AccessDesign of hypoxia responsive CRISPR-Cas9 for target gene regulation
- Yan An
- , Chandana S. Talwar
- & Eui-Jeon Woo
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| Open AccessCharacteristics of FXa-storing platelets in hemophilia B mice and the influence of alcohol on the platelets
- Wenwen Han
- , Rui Huang
- & Guowei Zhang
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| Open AccessHigh spontaneous integration rates of end-modified linear DNAs upon mammalian cell transfection
- Samuel Lim
- , R. Rogers Yocum
- & Jeffrey C. Way
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| Open AccessUpregulation of alveolar fluid clearance is not sufficient for Na+,K+-ATPase β subunit-mediated gene therapy of LPS-induced acute lung injury in mice
- Jing Liu
- , Gillian M. Schiralli-Lester
- & David A. Dean
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| Open AccessFull-length human dystrophin on human artificial chromosome compensates for mouse dystrophin deficiency in a Duchenne muscular dystrophy mouse model
- Yosuke Hiramuki
- , Satoshi Abe
- & Yasuhiro Kazuki
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| Open AccessCRISPR/Cas9 mediated specific ablation of vegfa in retinal pigment epithelium efficiently regresses choroidal neovascularization
- Jinkyu Park
- , Gang Cui
- & Suk Ho Byeon
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| Open AccessMachine learning and metabolic modelling assisted implementation of a novel process analytical technology in cell and gene therapy manufacturing
- Thomas Williams
- , Kevin Kalinka
- & Duygu Dikicioglu
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| Open AccessCRISPR-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
- Shih-hsin Kan
- , Jeffrey Y. Huang
- & Raymond Y. Wang
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| Open AccessEffects of pre-existing anti-adenovirus antibodies on transgene expression levels and therapeutic efficacies of arming oncolytic adenovirus
- Ryosuke Ono
- , Fumitaka Nishimae
- & Hiroyuki Mizuguchi
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| Open AccessArtificial intelligence workflow quantifying muscle features on Hematoxylin–Eosin stained sections reveals dystrophic phenotype amelioration upon treatment
- Marie Reinbigler
- , Jérémie Cosette
- & Daniel Stockholm
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| Open AccessOverexpression of VEGF in dermal fibroblast cells accelerates the angiogenesis and wound healing function: in vitro and in vivo studies
- Forough Shams
- , Hamideh Moravvej
- & Hamidreza Moosavian
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| Open AccessGenetic correction of haemoglobin E in an immortalised haemoglobin E/beta-thalassaemia cell line using the CRISPR/Cas9 system
- Kongtana Trakarnsanga
- , Nontaphat Thongsin
- & Methichit Wattanapanitch
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| Open AccessDelivery and assessment of a CRISPR/nCas9-based genome editing system on in vitro models of mucopolysaccharidoses IVA assisted by magnetite-based nanoparticles
- Andrés Felipe Leal
- , Javier Cifuentes
- & Carlos Javier Alméciga-Díaz
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| Open Accessp63 silencing induces epigenetic modulation to enhance human cardiac fibroblast to cardiomyocyte-like differentiation
- Jaya Pratap Pinnamaneni
- , Vivek P. Singh
- & Todd K. Rosengart
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| Open AccessMuscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription
- Amédée Mollard
- , Cécile Peccate
- & Stéphanie Lorain
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| Open AccessHideRNAs protect against CRISPR-Cas9 re-cutting after successful single base-pair gene editing
- Tim J. W. Harmsen
- , Colin E. J. Pritchard
- & Hein te Riele
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| Open AccessStent-based delivery of AAV2 vectors encoding oxidation-resistant apoA1
- Bahman Hooshdaran
- , Benjamin B. Pressly
- & Ilia Fishbein
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| Open AccessTransiently expressed CRISPR/Cas9 induces wild-type dystrophin in vitro in DMD patient myoblasts carrying duplications
- Veronica Pini
- , Virginie Mariot
- & Francesco Muntoni
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| Open AccessTumor RNA transfected DCs derived from iPS cells elicit cytotoxicity against cancer cells induced from colorectal cancer patients in vitro
- Shimpei Maruoka
- , Toshiyasu Ojima
- & Hiroki Yamaue
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| Open AccessTherapeutic potential of highly functional codon-optimized microutrophin for muscle-specific expression
- Anna V. Starikova
- , Victoria V. Skopenkova
- & Maryana V. Bardina
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| Open AccessRGEN-seq for highly sensitive amplification-free screen of off-target sites of gene editors
- Alexander Kuzin
- , Brendan Redler
- & Alexei Slesarev
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| Open AccessBlood phenylalanine reduction reverses gene expression changes observed in a mouse model of phenylketonuria
- Rachna Manek
- , Yao V. Zhang
- & Sirkka R. M. Kyostio-Moore
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| Open AccessFunctional roles of the membrane-associated AAV protein MAAP
- Lionel Galibert
- , Amira Hyvönen
- & Kari J. Airenne
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| Open AccessA combined treatment regimen of MGMT-modified γδ T cells and temozolomide chemotherapy is effective against primary high grade gliomas
- Lawrence S. Lamb
- , Larisa Pereboeva
- & H. Trent Spencer
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| Open AccessGene transfer of MRCKα rescues lipopolysaccharide-induced acute lung injury by restoring alveolar capillary barrier function
- Jing Liu
- & David A. Dean
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| Open AccessA guide in lentiviral vector production for hard-to-transfect cells, using cardiac-derived c-kit expressing cells as a model system
- V. Kalidasan
- , Wai Hoe Ng
- & Kumitaa Theva Das
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| Open AccessMesenchymal stromal cells as carriers of IL-12 reduce primary and metastatic tumors of murine melanoma
- Natalia Kułach
- , Ewelina Pilny
- & Ryszard Smolarczyk
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| Open AccessLentiviral and AAV-mediated expression of palivizumab offer protection against Respiratory Syncytial Virus infection
- Agata Antepowicz
- , Omar Habib
- & Stephen C. Hyde
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| Open AccessViral-mediated transduction of auditory neurons with opsins for optical and hybrid activation
- Rachael T. Richardson
- , Alex C. Thompson
- & James B. Fallon
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| Open AccessComprehensive characterization and quantification of adeno associated vectors by size exclusion chromatography and multi angle light scattering
- Nicole L. McIntosh
- , Geoffrey Y. Berguig
- & Vikas S. Bhat
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| Open AccessEpigenetic silencing directs expression heterogeneity of stably integrated multi-transcript unit genetic circuits
- Jan Zimak
- , Zachary W. Wagoner
- & Weian Zhao
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| Open AccessElectroporation outperforms in vivo-jetPEI for intratumoral DNA-based reporter gene transfer
- Liesl Jacobs
- , Elien De Smidt
- & Kevin Hollevoet
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| Open AccessHigh-throughput continuous-flow microfluidic electroporation of mRNA into primary human T cells for applications in cellular therapy manufacturing
- Charles A. Lissandrello
- , Jose A. Santos
- & Vishal Tandon
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| Open AccessGenome targeting by hybrid Flp-TAL recombinases
- Eugenia Voziyanova
- , Feng Li
- & Yuri Voziyanov
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| Open AccessRNA editing of BFP, a point mutant of GFP, using artificial APOBEC1 deaminase to restore the genetic code
- Sonali Bhakta
- , Matomo Sakari
- & Toshifumi Tsukahara
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| Open AccessViromers as carriers for mRNA-mediated expression of therapeutic molecules under inflammatory conditions
- Edith Jansig
- , Stefanie Geissler
- & Holger Cynis
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| Open AccessRapid repair of human disease-specific single-nucleotide variants by One-SHOT genome editing
- Yuji Yokouchi
- , Shinichi Suzuki
- & Takumi Era
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| Open AccessDNA unchained: two assays to discover and study inhibitors of the DNA clustering function of barrier-to-autointegration factor
- Michael Burger
- , Caroline Schmitt-Koopmann
- & Jean-Christophe Leroux
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| Open AccessTranscriptional repression of PTEN in neural cells using CRISPR/dCas9 epigenetic editing
- C. Moses
- , S. I. Hodgetts
- & A. R. Harvey
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| Open AccessComparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34+ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin
- Andrés Lamsfus-Calle
- , Alberto Daniel-Moreno
- & Markus Mezger
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| Open AccessAdeno-associated virus-binding antibodies detected in cats living in the Northeastern United States lack neutralizing activity
- Kei Adachi
- , Gregory A. Dissen
- & Hiroyuki Nakai
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| Open AccessDensity functional theory studies on cytosine analogues for inducing double-proton transfer with guanine
- Jinjie Xue
- , Xingping Guo
- & Yafeng Xiao
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| Open AccessTALEN mediated gene editing in a mouse model of Fanconi anemia
- Maria José Pino-Barrio
- , Yari Giménez
- & Susana Navarro
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| Open AccessSpecificity profiling of CRISPR system reveals greatly enhanced off-target gene editing
- Yao Wang
- , Mingrui Wang
- & Quan Du