Featured
-
-
Article
| Open Access
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
- Shih-hsin Kan
- , Jeffrey Y. Huang
- & Raymond Y. Wang
-
Article
| Open Access
HideRNAs protect against CRISPR-Cas9 re-cutting after successful single base-pair gene editing
- Tim J. W. Harmsen
- , Colin E. J. Pritchard
- & Hein te Riele
-
Article
| Open Access
Transiently expressed CRISPR/Cas9 induces wild-type dystrophin in vitro in DMD patient myoblasts carrying duplications
- Veronica Pini
- , Virginie Mariot
- & Francesco Muntoni
-
Article
| Open Access
RGEN-seq for highly sensitive amplification-free screen of off-target sites of gene editors
- Alexander Kuzin
- , Brendan Redler
- & Alexei Slesarev
-
Article
| Open Access
Genome targeting by hybrid Flp-TAL recombinases
- Eugenia Voziyanova
- , Feng Li
- & Yuri Voziyanov
-
Article
| Open Access
Rapid repair of human disease-specific single-nucleotide variants by One-SHOT genome editing
- Yuji Yokouchi
- , Shinichi Suzuki
- & Takumi Era
-
Article
| Open Access
Density functional theory studies on cytosine analogues for inducing double-proton transfer with guanine
- Jinjie Xue
- , Xingping Guo
- & Yafeng Xiao
-
Article
| Open Access
TALEN mediated gene editing in a mouse model of Fanconi anemia
- Maria José Pino-Barrio
- , Yari Giménez
- & Susana Navarro
-
Article
| Open Access
Specificity profiling of CRISPR system reveals greatly enhanced off-target gene editing
- Yao Wang
- , Mingrui Wang
- & Quan Du
-
Article
| Open Access
The R229Q mutation of Rag2 does not characterize severe immunodeficiency in mice
- Young Jin
- , Ara Lee
- & Sang-Jun Ha
-
Article
| Open Access
A note on retrograde gene transfer efficiency and inflammatory response of lentiviral vectors pseudotyped with FuG-E vs. FuG-B2 glycoproteins
- Soshi Tanabe
- , Shiori Uezono
- & Masahiko Takada
-
Article
| Open Access
Engineering of Primary Human B cells with CRISPR/Cas9 Targeted Nuclease
- Matthew J. Johnson
- , Kanut Laoharawee
- & Branden S. Moriarity
-
Article
| Open Access
HIV-1 inhibition in cells with CXCR4 mutant genome created by CRISPR-Cas9 and piggyBac recombinant technologies
- Shuai Liu
- , Qiankun Wang
- & Shuliang Chen
-
Article
| Open Access
Small nuclear RNA-mediated modulation of splicing reveals a therapeutic strategy for a TREM2 mutation and its post-transcriptional regulation
- Motoaki Yanaizu
- , Kenji Sakai
- & Jun-ichi Satoh
-
Article
| Open Access
Delivery of exogenous mitochondria via centrifugation enhances cellular metabolic function
- Mi Jin Kim
- , Jung Wook Hwang
- & Yong-Soo Choi
-
Article
| Open Access
Deep mutational scanning of S. pyogenes Cas9 reveals important functional domains
- Jeffrey M. Spencer
- & Xiaoliu Zhang
-
Article
| Open Access
Gene delivery of hypoxia-inducible VEGF targeting collagen effectively improves cardiac function after myocardial infarction
- Jing-Bo Xia
- , Hai-Yan Wu
- & Xu-Feng Qi
-
Article
| Open Access
In vivo genetic manipulation of inner ear connexin expression by bovine adeno-associated viral vectors
- Giulia Crispino
- , Fabian Galindo Ramirez
- & Fabio Mammano
-
Article
| Open Access
Gene-based Therapy in a Mouse Model of Blue Cone Monochromacy
- Yuxin Zhang
- , Wen-Tao Deng
- & Ji-jing Pang
-
Article
| Open Access
CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice
- Tsukasa Ohmori
- , Yasumitsu Nagao
- & Yoichi Sakata
-
Article
| Open Access
Reprogramming MHC specificity by CRISPR-Cas9-assisted cassette exchange
- William Kelton
- , Ann Cathrin Waindok
- & Sai T. Reddy
-
Article
| Open Access
Deregulation of the miR-16-KRAS axis promotes colorectal cancer
- Chaoying You
- , Hongwei Liang
- & Yi Ba
-
Article
| Open Access
Metabolic flux profiling of MDCK cells during growth and canine adenovirus vector production
- Nuno Carinhas
- , Daniel A. M. Pais
- & Ana P. Teixeira
-
Article
| Open Access
AAV Gene Therapy for MPS1-associated Corneal Blindness
- Melisa Vance
- , Telmo Llanga
- & Matthew L. Hirsch
-
Article
| Open Access
In situ genetic correction of F8 intron 22 inversion in hemophilia A patient-specific iPSCs
- Yong Wu
- , Zhiqing Hu
- & Desheng Liang
-
Article
| Open Access
Controlled delivery of β-globin-targeting TALENs and CRISPR/Cas9 into mammalian cells for genome editing using microinjection
- Renee N. Cottle
- , Ciaran M. Lee
- & Gang Bao
-
Article
| Open Access
Genome editing of CXCR4 by CRISPR/cas9 confers cells resistant to HIV-1 infection
- Panpan Hou
- , Shuliang Chen
- & Deyin Guo
-
Article
| Open Access
Novel lentiviral vectors with mutated reverse transcriptase for mRNA delivery of TALE nucleases
- Ulrike Mock
- , Kristoffer Riecken
- & Boris Fehse
-
Article
| Open Access
Transcription activator-like effector nuclease-mediated transduction of exogenous gene into IL2RG locus
- Yohei Matsubara
- , Tomoki Chiba
- & Hiroshi Asahara
-
Article
| Open Access
Non-integrating gamma-retroviral vectors as a versatile tool for transient zinc-finger nuclease delivery
- Sylwia Bobis-Wozowicz
- , Melanie Galla
- & Toni Cathomen
-
Article
| Open Access
Development of novel DNA vaccine for VEGF in murine cancer model
- Mariko Kyutoku
- , Hironori Nakagami
- & Ryuichi Morishita
-
Article
| Open Access
Expanded activity of dimer nucleases by combining ZFN and TALEN for genome editing
- Wei Yan
- , Cory Smith
- & Linzhao Cheng
Cardiac vein retroinjections provide an efficient approach for global left ventricular gene transfer with adenovirus and adeno-associated virus