Journal ArticleDOI
AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway.
Pradeep S. B. Podila,Shun-Qing Liang,Christopher J. Walkey,Alexa E. Martinez,Qin Su,Mary E. Dickinson,Dan Wang,William R. Lagor,Jason D. Heaney,Guangping Gao,Wen Xue +10 more
TLDR
In this article, a dual adeno-associated virus (AAV) CRISPR platform was developed and validated for effective editing of a lox-stop-lox-Tomato reporter in mouse lung airway.About:
This article is published in Molecular Therapy.The article was published on 2021-10-23. It has received 16 citations till now. The article focuses on the topics: Genome editing & CRISPR.read more
Citations
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Journal ArticleDOI
Progress of delivery methods for CRISPR-Cas9
TL;DR: The development of delivery methods is highly likely to take the application of CRISPR-Cas9 technology to another level, and the complex nuclease and protease environment is a tremendous challenge for in vivo clinical applications.
Journal ArticleDOI
rAAV-MEDIATED EDITING OF THE G551D CFTR MUTATION IN FERRET AIRWAY BASAL CELLS.
Ziying Yan,Kai Vorhies,Zehua Feng,Soo Yeun Park,Soon H. Choi,Yulong Zhang,Micheal Winter,Xingshen Sun,John F. Engelhardt +8 more
TL;DR:
Journal ArticleDOI
In vivo delivery of CRISPR-Cas9 genome editing components for therapeutic applications.
TL;DR: In this article , the authors highlight the therapeutic applications of in vivo genome editing mediated by the CRISPR-Cas9 system and highlight the challenges and opportunities in the field of in-vivo genome editing.
Journal ArticleDOI
Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing
Rajith S. Manan,Shunqing Liang,Akiva G. R. Gordon,A. Jiang,Andrew J. Varley,Guangping Gao,Robert Langer,Wen Xue,Daniel G. Anderson +8 more
Journal ArticleDOI
AAV-mediated gene editing lights up the lung
TL;DR: Liang et al. as mentioned in this paper demonstrated efficient CRISPR-based gene editing in both the large and small airways (∼16%−26%) using a dual-recombinant adeno-associated viral (rAAV) vector approach that activated a fluorescent reporter by targeted deletion of a LoxP-STOP-LoxP sequence within transgenic mice.
References
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A robust and high-throughput Cre reporting and characterization system for the whole mouse brain
Linda Madisen,Theresa A. Zwingman,Susan M. Sunkin,Seung Wook Oh,Hatim A. Zariwala,Hong Gu,Lydia Ng,Richard D. Palmiter,Michael Hawrylycz,Allan R. Jones,Ed S. Lein,Hongkui Zeng +11 more
TL;DR: A set of Cre reporter mice with strong, ubiquitous expression of fluorescent proteins of different spectra is generated and enables direct visualization of fine dendritic structures and axonal projections of the labeled neurons, which is useful in mapping neuronal circuitry, imaging and tracking specific cell populations in vivo.
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The new frontier of genome engineering with CRISPR-Cas9
TL;DR: The power of the CRISPR-Cas9 technology to systematically analyze gene functions in mammalian cells, study genomic rearrangements and the progression of cancers or other diseases, and potentially correct genetic mutations responsible for inherited disorders is illustrated.
Journal ArticleDOI
Basal cells as stem cells of the mouse trachea and human airway epithelium
Jason R. Rock,Mark W. Onaitis,Emma L. Rawlins,Yun Lu,Cheryl P. Clark,Yan Xue,Scott H. Randell,Brigid L.M. Hogan +7 more
TL;DR: The pseudostratified epithelium of the mouse trachea and human airways contains a population of basal cells expressing Trp-63 and cytokeratins 5, which generate differentiated cells during postnatal growth and in the adult during both steady state and epithelial repair.
Journal ArticleDOI
Adeno-associated virus vector as a platform for gene therapy delivery
TL;DR: The fundamentals of AAV and vectorology are discussed, focusing on current therapeutic strategies, clinical progress and ongoing challenges.
Journal ArticleDOI
In vivo gene editing in dystrophic mouse muscle and muscle stem cells
Mohammadsharif Tabebordbar,Kexian Zhu,Jason Cheng,Wei Leong Chew,Jeffrey J. Widrick,Winston X. Yan,Winston X. Yan,Claire Maesner,Elizabeth Y Wu,Ru Xiao,F. Ann Ran,F. Ann Ran,Le Cong,Le Cong,Feng Zhang,Feng Zhang,Luk H. Vandenberghe,George M. Church,Amy J. Wagers +18 more
TL;DR: In this paper, the authors developed and tested a direct gene-editing approach to induce exon deletion and recover dystrophin expression in the mdx mouse model of Duchenne muscular dystrophy.