Literature DB >> 28263568

In Vivo Delivery of CRISPR/Cas9 for Therapeutic Gene Editing: Progress and Challenges.

Rubul Mout1, Moumita Ray1, Yi-Wei Lee1, Federica Scaletti1, Vincent M Rotello1.   

Abstract

The successful use of clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9-based gene editing for therapeutics requires efficient in vivo delivery of the CRISPR components. There are, however, major challenges on the delivery front. In this Topical Review, we will highlight recent developments in CRISPR delivery, and we will present hurdles that still need to be overcome to achieve effective in vivo editing.

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Year:  2017        PMID： 28263568      PMCID： PMC5846329          DOI： 10.1021/acs.bioconjchem.7b00057

Source DB:  PubMed          Journal:  Bioconjug Chem        ISSN： 1043-1802            Impact factor:   4.774


  39 in total

Review 1.  Towards a systems understanding of MHC class I and MHC class II antigen presentation.

Authors:  Jacques Neefjes; Marlieke L M Jongsma; Petra Paul; Oddmund Bakke
Journal:  Nat Rev Immunol       Date:  2011-11-11       Impact factor: 53.106

Review 2.  Delivery and Specificity of CRISPR-Cas9 Genome Editing Technologies for Human Gene Therapy.

Authors:  Jennifer L Gori; Patrick D Hsu; Morgan L Maeder; Shen Shen; G Grant Welstead; David Bumcrot
Journal:  Hum Gene Ther       Date:  2015-07       Impact factor: 5.695

Review 3.  Genome editing. The new frontier of genome engineering with CRISPR-Cas9.

Authors:  Jennifer A Doudna; Emmanuelle Charpentier
Journal:  Science       Date:  2014-11-28       Impact factor: 47.728

4.  Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease.

Authors:  Seung Woo Cho; Sojung Kim; Jong Min Kim; Jin-Soo Kim
Journal:  Nat Biotechnol       Date:  2013-01-29       Impact factor: 54.908

5.  In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

Authors:  Christopher E Nelson; Chady H Hakim; David G Ousterout; Pratiksha I Thakore; Eirik A Moreb; Ruth M Castellanos Rivera; Sarina Madhavan; Xiufang Pan; F Ann Ran; Winston X Yan; Aravind Asokan; Feng Zhang; Dongsheng Duan; Charles A Gersbach
Journal:  Science       Date:  2015-12-31       Impact factor: 47.728

6.  Characterization of genome integrity for oversized recombinant AAV vector.

Authors:  Biao Dong; Hiroyuki Nakai; Weidong Xiao
Journal:  Mol Ther       Date:  2009-11-10       Impact factor: 11.454

7.  Effect of genome size on AAV vector packaging.

Authors:  Zhijian Wu; Hongyan Yang; Peter Colosi
Journal:  Mol Ther       Date:  2009-11-10       Impact factor: 11.454

8.  Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing.

Authors:  Qiurong Ding; Alanna Strong; Kevin M Patel; Sze-Ling Ng; Bridget S Gosis; Stephanie N Regan; Chad A Cowan; Daniel J Rader; Kiran Musunuru
Journal:  Circ Res       Date:  2014-06-10       Impact factor: 17.367

Review 9.  Nanocarriers as an emerging platform for cancer therapy.

Authors:  Dan Peer; Jeffrey M Karp; Seungpyo Hong; Omid C Farokhzad; Rimona Margalit; Robert Langer
Journal:  Nat Nanotechnol       Date:  2007-12       Impact factor: 39.213

10.  In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa.

Authors:  Benjamin Bakondi; Wenjian Lv; Bin Lu; Melissa K Jones; Yuchun Tsai; Kevin J Kim; Rachelle Levy; Aslam Abbasi Akhtar; Joshua J Breunig; Clive N Svendsen; Shaomei Wang
Journal:  Mol Ther       Date:  2015-12-15       Impact factor: 11.454
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  53 in total

1.  General Strategy for Direct Cytosolic Protein Delivery via Protein-Nanoparticle Co-engineering.

Authors:  Rubul Mout; Moumita Ray; Tristan Tay; Kanae Sasaki; Gulen Yesilbag Tonga; Vincent M Rotello
Journal:  ACS Nano       Date:  2017-06-15       Impact factor: 15.881

Review 2.  Approach for in vivo delivery of CRISPR/Cas system: a recent update and future prospect.

Authors:  Yu-Fan Chuang; Andrew J Phipps; Fan-Li Lin; Valerie Hecht; Alex W Hewitt; Peng-Yuan Wang; Guei-Sheung Liu
Journal:  Cell Mol Life Sci       Date:  2021-01-03       Impact factor: 9.261

Review 3.  Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications.

Authors:  Chang Liu; Li Zhang; Hao Liu; Kun Cheng
Journal:  J Control Release       Date:  2017-09-11       Impact factor: 9.776

4.  CRISPRed Macrophages for Cell-Based Cancer Immunotherapy.

Authors:  Moumita Ray; Yi-Wei Lee; Joseph Hardie; Rubul Mout; Gulen Yeşilbag Tonga; Michelle E Farkas; Vincent M Rotello
Journal:  Bioconjug Chem       Date:  2018-01-22       Impact factor: 4.774

Review 5.  [Development of CRISPR technology and its application in bone and cartilage tissue engineering].

Authors:  Guo Chen; Du Cheng; Bin Chen
Journal:  Nan Fang Yi Ke Da Xue Xue Bao       Date:  2019-12-30

Review 6.  Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects.

Authors:  Hongyi Li; Yang Yang; Weiqi Hong; Mengyuan Huang; Min Wu; Xia Zhao
Journal:  Signal Transduct Target Ther       Date:  2020-01-03

7.  Nonviral Nanoparticles for CRISPR-Based Genome Editing: Is It Just a Simple Adaption of What Have Been Developed for Nucleic Acid Delivery?

Authors:  Min Qiu; Zachary Glass; Qiaobing Xu
Journal:  Biomacromolecules       Date:  2019-08-07       Impact factor: 6.988

Review 8.  Nanotechnology approaches to eradicating HIV reservoirs.

Authors:  Shijie Cao; Kim A Woodrow
Journal:  Eur J Pharm Biopharm       Date:  2018-06-04       Impact factor: 5.571

9.  In Vivo Editing of Macrophages through Systemic Delivery of CRISPR-Cas9-Ribonucleoprotein-Nanoparticle Nanoassemblies.

Authors:  Yi-Wei Lee; Rubul Mout; David C Luther; Yuanchang Liu; Laura Castellanos-García; Amy S Burnside; Moumita Ray; Gulen Yeşilbag Tonga; Joseph Hardie; Harini Nagaraj; Riddha Das; Erin L Phillips; Tristan Tay; Richard W Vachet; Vincent M Rotello
Journal:  Adv Ther (Weinh)       Date:  2019-08-15

10.  Delivery of Tissue-Targeted Scalpels: Opportunities and Challenges for In Vivo CRISPR/Cas-Based Genome Editing.

Authors:  Tuo Wei; Qiang Cheng; Lukas Farbiak; Daniel G Anderson; Robert Langer; Daniel J Siegwart
Journal:  ACS Nano       Date:  2020-07-22       Impact factor: 15.881
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