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Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo
- Neda A. M. Nafchi
- , Ellie M. Chilcott
- & Rafael J. Yáñez-Muñoz
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Tolerability and tropism of recombinant adeno-associated virus vectors in the African green monkey (Chlorocebus sabaeus) anterior chamber
- Kristina J. Chern
- , Kimicia Z. Issac
- & Daniel M. Lipinski
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Article
| Open AccessTerminal hairpins improve protein expression in IRES-initiated mRNA in the absence of a cap and polyadenylated tail
- Victor Solodushko
- & Brian Fouty
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An ectopic enhancer restores CFTR expression through de novo chromatin looping
- Jenny L. Kerschner
- , Alekh Paranjapye
- & Ann Harris
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Activation of homology-directed DNA repair plays key role in CRISPR-mediated genome correction
- Gourish Mondal
- , Caitlin J. VanLith
- & Joseph B. Lillegard
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Review Article
| Open AccessSplicing mutations in the CFTR gene as therapeutic targets
- Karine Deletang
- & Magali Taulan-Cadars
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Article |
Forced activation of dystrophin transcription by CRISPR/dCas9 reduced arrhythmia susceptibility via restoring membrane Nav1.5 distribution
- Ruixin Zhang
- , Junwu Liu
- & Zhenwei Pan
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Review Article |
Prospects of viral vector-mediated delivery of sequences encoding anti-HBV designer endonucleases
- Ridhwaanah Jacobs
- , Prashika Singh
- & Mohube Betty Maepa
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Article
| Open AccessExon skipping induced by CRISPR-directed gene editing regulates the response to chemotherapy in non-small cell lung carcinoma cells
- Kelly Banas
- , Shirin Modarai
- & Eric B. Kmiec
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Article |
Epigenetic repression of gonadotropin gene expression via a GnRH-mediated DNA delivery system
- Lilach Pnueli
- & Philippa Melamed
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CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing
- Pouiré Yaméogo
- , Benjamin L. Duchêne
- & Jacques P. Tremblay
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Article |
Novel design of nucleic acid standards for hydrolysis probe-based PCR with melting analysis
- Anna Baoutina
- & Somanath Bhat
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Brief Communication
| Open AccessAlternative oxidase encoded by sequence-optimized and chemically-modified RNA transfected into mammalian cells is catalytically active
- Luca Giordano
- , Manish K. Aneja
- & Marten Szibor
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Article
| Open AccessHighly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice
- Sam Hana
- , Michael Peterson
- & Shih-Ching Lo
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Brief Communication
| Open AccessDevelopment of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor
- Elena Barbon
- , Charlotte Kawecki
- & Federico Mingozzi
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Article |
FEN1 inhibitor synergizes with low-dose camptothecin to induce increased cell killing via the mitochondria mediated apoptotic pathway
- Ting Wu
- , Hongqiao Zhu
- & Zhigang Guo
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Review Article |
Gene editing and RNAi approaches for COVID-19 diagnostics and therapeutics
- Burak Berber
- , Cihan Aydin
- & Cihan Tastan
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Article |
Adenovirus vector-mediated YKL-40 shRNA attenuates eosinophil airway inflammation in a murine asthmatic model
- Ling Wang
- , Aihua Bao
- & Suqin Ben
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Brief Communication
| Open AccessPrecise and error-prone CRISPR-directed gene editing activity in human CD34+ cells varies widely among patient samples
- Shirin R. Modarai
- , Sambee Kanda
- & Eric B. Kmiec
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Promoting role of circ-Jarid2/miR-129-5p/Celf1 axis in cardiac hypertrophy
- Yan Fang
- , Yu Tao
- & Hengli Lai
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Article
| Open AccessMaximizing lentiviral vector gene transfer in the CNS
- Morgane Humbel
- , Mergim Ramosaj
- & Nicole Déglon
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miR-503-5p inhibits colon cancer tumorigenesis, angiogenesis, and lymphangiogenesis by directly downregulating VEGF-A
- Linlin Wei
- , Chaonan Sun
- & Shichen Sun
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Article |
LncRNA SNHG16 induces proliferation and fibrogenesis via modulating miR-141-3p and CCND1 in diabetic nephropathy
- Xiaohong Jiang
- , Qianying Ru
- & Shan Huang
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Review Article |
Recent development of AAV-based gene therapies for inner ear disorders
- Yiyang Lan
- , Yong Tao
- & Guisheng Zhong
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Article |
Sigma-1 receptor activity in primary sensory neurons is a critical driver of neuropathic pain
- Seung Min Shin
- , Fei Wang
- & Hongwei Yu
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Review Article |
Advances in genome editing: the technology of choice for precise and efficient β-thalassemia treatment
- Gibran Ali
- , Muhammad Akram Tariq
- & Javed Akram
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Comment |
Treating osteoarthritis via gene therapy with rejuvenation factors
- Jiahe Zhang
- , Guang-Hui Liu
- & Moshi Song
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Brief Communication |
‘Mini’ U6 Pol III promoter exhibits nucleosome redundancy and supports multiplexed coupling of CRISPR/Cas9 effects
- Roland Preece
- , Christos Georgiadis
- & Waseem Qasim
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Article
| Open AccessNr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa
- Sujun Li
- , Shyamtanu Datta
- & Neena B. Haider
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Article |
CRISPR/Cas9 increases mitotic gene conversion in human cells
- Parisa Javidi-Parsijani
- , Pin Lyu
- & Baisong Lu
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Article |
The deletion of mutant SOD1 via CRISPR/Cas9/sgRNA prolongs survival in an amyotrophic lateral sclerosis mouse model
- Weisong Duan
- , Moran Guo
- & Chunyan Li
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Article
| Open AccessTransduction optimization of AAV vectors for human gene therapy of glaucoma and their reversed cell entry characteristics
- Laura Rodriguez-Estevez
- , Priyadarsini Asokan
- & Teresa Borrás
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Article |
MiR-21 derived from the exosomes of MSCs regulates the death and differentiation of neurons in patients with spinal cord injury
- Jian Kang
- , Zhenhuan Li
- & Guanghui Xu
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Article |
Estrogen administration reduces the risk of pulmonary arterial hypertension by modulating the miR-133a signaling pathways in rats
- Sijing Zhou
- , Ke Zhu
- & Ran Wang
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Brief Communication |
Design and applications of gene therapy vectors for mucopolysaccharidosis in Colombia
- Carlos J. Alméciga-Diaz
- & Luis A. Barrera
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Comment |
Bringing balance to the force–regulatable gene therapy for epilepsy
- Arnaud J. Ruiz
- & Ahad A. Rahim
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Article |
Polarized AAVR expression determines infectivity by AAV gene therapy vectors
- Bradley A. Hamilton
- , Xiaopeng Li
- & Joseph Zabner
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Article |
ILK promotes survival and self-renewal of hypoxic MSCs via the activation of lncTCF7-Wnt pathway induced by IL-6/STAT3 signaling
- Qing Mao
- , Xiu-Lin Liang
- & Yong-Xiang Lu
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Article |
Histone deacetylase inhibitors reactivate silenced transgene in vivo
- Chunbo Zhang
- , Guisheng Zhang
- & Dexi Liu
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Original Article |
Site-directed RNA editing by adenosine deaminase acting on RNA for correction of the genetic code in gene therapy
- Md T A Azad
- , S Bhakta
- & T Tsukahara
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Original Article |
Targeting the CXCR4/CXCL12 axis in treating epithelial ovarian cancer
- T L Mao
- , K F Fan
- & C L Liu
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Original Article |
Nasal application of HSV encoding human preproenkephalin blocks craniofacial pain in a rat model of traumatic brain injury
- A C Meidahl
- , M Klukinov
- & D C Yeomans
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Original Article |
miRNA-mediated post-transcriptional silencing of transgenes leads to increased adeno-associated viral vector yield and targeting specificity
- C A Reid
- , S L Boye
- & D M Lipinski
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Original Article |
Silencing of HIV-1 by AgoshRNA molecules
- E Herrera-Carrillo
- , A Harwig
- & B Berkhout
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Original Article |
Synergistic effects of deleting multiple nonessential elements in nonreplicative HSV-1 BAC genomic vectors play a critical role in their viability
- M Ventosa
- , A Ortiz-Temprano
- & F Lim
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Review |
How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy
- N N Singh
- , M D Howell
- & R N Singh
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Original Article |
Potential mechanisms of microRNA-141-3p to alleviate chronic inflammatory pain by downregulation of downstream target gene HMGB1: in vitro and in vivo studies
- W-S Shen
- , X-Q Xu
- & Y-H Yu
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