Engineered Viruses as Genome Editing Devices

Molecular Therapy : the Journal of the American Society of Gene Therapy
Xiaoyu Chen, Manuel A F V Gonçalves

Abstract

Genome editing based on sequence-specific designer nucleases, also known as programmable nucleases, seeks to modify in a targeted and precise manner the genetic information content of living cells. Delivering into cells designer nucleases alone or together with donor DNA templates, which serve as surrogate homologous recombination (HR) substrates, can result in gene knockouts or gene knock-ins, respectively. As engineered replication-defective viruses, viral vectors are having an increasingly important role as delivery vehicles for donor DNA templates and designer nucleases, namely, zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered, regularly interspaced, short palindromic repeats (CRISPR)-associated Cas9 (CRISPR-Cas9) nucleases, also known as RNA-guided nucleases (RGNs). We review this dual role played by engineered viral particles on genome editing while focusing on their main scaffolds, consisting of lentiviruses, adeno-associated viruses, and adenoviruses. In addition, the coverage of the growing body of research on the repurposing of viral vectors as delivery systems for genome editing tools is complemented with information regarding their main characteristics, pros, and c...Continue Reading

References

Jun 21, 1994·Proceedings of the National Academy of Sciences of the United States of America·P RouetM Jasin
Apr 16, 1998·Nature Genetics·D W Russell, R K Hirata
Feb 23, 2000·Journal of Virology·D M ShayakhmetovA Lieber
Oct 9, 2001·Human Gene Therapy·M KumarR E Sutton
Jun 29, 2002·Nature Biotechnology·Roli HirataDavid W Russell
Aug 23, 2002·Proceedings of the National Academy of Sciences of the United States of America·Guang-Ping GaoJames M Wilson
May 2, 2003·Molecular and Cellular Biology·Daniel G MillerDavid W Russell
May 2, 2003·Molecular and Cellular Biology·Matthew H PorteusDavid Baltimore
Jun 23, 2004·Nature Genetics·Daniel G MillerDavid W Russell
Jul 27, 2004·Nature Medicine·Paul GregorevicJeffrey S Chamberlain
Dec 1, 2004·Annual Review of Genetics·Douglas M McCartyR Jude Samulski
Apr 16, 2005·BioEssays : News and Reviews in Molecular, Cellular and Developmental Biology·Manuel A F V Gonçalves
May 10, 2005·Virology Journal·Manuel A F V Gonçalves
May 26, 2005·The Journal of General Virology·Jort VellingaRob C Hoeben
Aug 17, 2005·Current Gene Therapy·James CroninJakob Reiser
Sep 22, 2005·Proceedings of the National Academy of Sciences of the United States of America·Fumi OhbayashiKohnosuke Mitani
Nov 2, 2005·Nature Reviews. Microbiology·Ana Vasileva, Rolf Jessberger
Jan 31, 2006·Molecular Therapy : the Journal of the American Society of Gene Therapy·Manuel A F V GonçalvesAntoine A F de Vries
May 20, 2006·Reviews in Medical Virology·Manuel A F V Gonçalves, Antoine A F de Vries
Aug 1, 2006·Nature Biotechnology·Daniel G MillerDavid W Russell
May 26, 2007·Human Gene Therapy·Nicola J Philpott, Adrian J Thrasher
Oct 4, 2007·Molecular Therapy : the Journal of the American Society of Gene Therapy·Florian Kreppel, Stefan Kochanek
Mar 6, 2008·Nature Reviews. Genetics·Siobain DuffyEdward C Holmes
Jun 6, 2009·Molecular Therapy : the Journal of the American Society of Gene Therapy·Klaus Wanisch, Rafael J Yáñez-Muñoz
Sep 29, 2009·Expert Opinion on Biological Therapy·Sergey S Seregin, Andrea Amalfitano
Oct 22, 2009·Proceedings of the National Academy of Sciences of the United States of America·Boris KantorTal Kafri
Jan 21, 2010·Molecular Therapy : the Journal of the American Society of Gene Therapy·Janka MátraiThierry VandenDriessche
Aug 10, 2010·FEBS Letters·Elizabeth M Kass, Maria Jasin
Mar 30, 2011·Nature Reviews. Genetics·Luigi Naldini
May 3, 2011·Human Molecular Genetics·Robert M Kotin
May 21, 2011·Cell Stem Cell·Guang-Hui LiuJuan Carlos Izpisua Belmonte
Aug 9, 2011·Nature Biotechnology·Richard GabrielChristof von Kalle
Nov 24, 2011·Molecular Therapy : the Journal of the American Society of Gene Therapy·Prashanth AsuriDavid V Schaffer
Dec 8, 2011·Molecular Therapy : the Journal of the American Society of Gene Therapy·Emi AizawaKohnosuke Mitani
Jan 19, 2012·Molecular Therapy : the Journal of the American Society of Gene Therapy·Luca BiascoAlessandro Aiuti
Feb 7, 2012·Cell Stem Cell·Sergei DoulatovJohn E Dick
Jan 1, 2013·Nature Reviews. Drug Discovery·Harald von Boehmer, Carolin Daniel

❮ Previous
Next ❯

Citations

Feb 8, 2016·Trends in Microbiology·Aryn A PriceDavid S Weiss
Apr 20, 2016·Frontiers in Genetics·Han B LeeKarl J Clark
May 7, 2016·Neurotherapeutics : the Journal of the American Society for Experimental NeuroTherapeutics·Martyn K WhiteKamel Khalili
May 25, 2016·Genome Medicine·Ignazio MaggioManuel A F V Gonçalves
Nov 21, 2016·Journal of Controlled Release : Official Journal of the Controlled Release Society·Jia Liu, Sai-Lan Shui
Oct 27, 2016·Clinical Pharmacology and Therapeutics·M P Calos
Jan 26, 2017·Plasmid·Maysam MansouriPhilipp Berger
Sep 9, 2016·Molecular Therapy. Methods & Clinical Development·Arnold ParkBenhur Lee
Mar 2, 2017·Histochemistry and Cell Biology·Andrea PeterStefan Hippenstiel
Jan 19, 2018·Biotechnology Letters·Kul BhushanDharmendra Pratap
Mar 27, 2018·The Journal of Gene Medicine·Samantha L GinnMohammad R Abedi
Jan 5, 2019·Advanced Pharmaceutical Bulletin·Hadi BayatAzam Rahimpour
May 23, 2019·Human Genetics·Minyoung Lee, Hyongbum Kim
Jun 15, 2019·Journal of Drug Targeting·Stefanie A PenaAdrien A Eshraghi
May 5, 2020·Journal of Materials Chemistry. B, Materials for Biology and Medicine·Yan GongShubiao Zhang
Feb 4, 2016·Oncotarget·Martyn K White, Kamel Khalili
Jun 9, 2020·Journal of Inherited Metabolic Disease·Hidde A ZittersteijnRob C Hoeben
Mar 25, 2017·Nature Reviews. Drug Discovery·Hao YinDaniel G Anderson
May 29, 2018·Drug Delivery·Christopher A LinoJerilyn A Timlin
Dec 6, 2018·Nature Reviews. Rheumatology·Magali Cucchiarini, Henning Madry
Feb 14, 2020·Briefings in Functional Genomics·Xing ChengXianfa Du
Apr 5, 2019·Nature Biomedical Engineering·Mu-Nung Hsu, Yu-Chen Hu
Jun 27, 2019·The Journal of Gene Medicine·Xiaojie XuYuan Ping
Dec 5, 2019·Wiley Interdisciplinary Reviews. Nanomedicine and Nanobiotechnology·Fengqian ChenQi Liu
Dec 22, 2016·Cell and Tissue Research·Andreas C HockeStefan Hippenstiel
Dec 1, 2018·Cellular and Molecular Life Sciences : CMLS·Shipra MalikRaman Bahal
Feb 18, 2017·Cellular and Molecular Life Sciences : CMLS·Kamel KhaliliJeffrey M Jacobson
Aug 5, 2018·Neurological Sciences : Official Journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology·Joshua KuruvillaAnna Pick Kiong Ling
Mar 5, 2019·Biological & Pharmaceutical Bulletin·Yağız Anıl ÇiçekVincent M Rotello
Nov 16, 2017·Current Protocols in Stem Cell Biology·Ravi Chandra YadaCynthia E Dunbar
Dec 31, 2020·Wound Repair and Regeneration : Official Publication of the Wound Healing Society [and] the European Tissue Repair Society·Calver PangMallappa K Kolar
Oct 20, 2020·Biochimica Et Biophysica Acta. Reviews on Cancer·Jie WangJinming Li
Jan 23, 2021·Viruses·Carla Mr VarandaPatrick Materatski
Jan 8, 2021·Virology Journal·César Augusto Diniz XavierAnna Elizabeth Whitfield
Jan 6, 2021·Nucleic Acids Research·Qian WangManuel A F V Gonçalves
Jul 9, 2020·Molecular Therapy. Methods & Clinical Development·Pedro R L PerdigãoJoao Goncalves
Mar 3, 2021·Proceedings of the National Academy of Sciences of the United States of America·Min QiuQiaobing Xu
May 23, 2020·Clinics in Laboratory Medicine·Elan Hahn, Matthew Hiemenz

❮ Previous
Next ❯

Methods Mentioned

BETA
gene
gene knock-in
genetic modification
transgenic
gene knockouts

Related Concepts

Related Feeds

CRISPR (general)

Clustered regularly interspaced short palindromic repeats (CRISPR) are DNA sequences in the genome that are recognized and cleaved by CRISPR-associated proteins (Cas). CRISPR-Cas system enables the editing of genes to create or correct mutations. Discover the latest research on CRISPR here.

Cell-Type-Specific Viral Vectors (ASM)

Viral vectors are used in biological research and therapy to deliver genetic material into cells. However, the efficiency of viral vectors varies depending on the cell type. Here is the latest research on cell-type-specific viral vectors.

Virology & CRISPR

This feed focuses on the virology of CRISPR and its use in developing CRISPR-Cas systems. Discover the latest research here.

Blood Clotting Disorders

Thrombophilia includes conditions with increased tendency for excessive blood clotting. Blood clotting occurs when the body has insufficient amounts of specialized proteins that make blood clot and stop bleeding. Here is the latest research on blood clotting disorders.

CRISPR for Genome Editing

Genome editing technologies enable the editing of genes to create or correct mutations. Clustered regularly interspaced short palindromic repeats (CRISPR) are DNA sequences in the genome that are recognized and cleaved by CRISPR-associated proteins (Cas). Here is the latest research on the use of CRISPR-Cas system in gene editing.

Cell-Type-Specific Viral Vectors

Viral vectors are used in biological research and therapy to deliver genetic material into cells. However, the efficiency of viral vectors varies depending on the cell type. Here is the latest research on cell-type-specific viral vectors.

AAV-based Gene Therapy

Adeno-associated virus (AAV)-based gene therapy is a biological vector that is being researched to be used as a potential therapeutic option. This gene therapy is designed to insert fragments of DNA into targeted cells to help treat diseases, such as hemophilia a. Discover the latest research on AAV-based gene therapy here.

CRISPR Ribonucleases Deactivation

CRISPR-Cas system enables the editing of genes to create or correct mutations. This feed focuses on mechanisms that underlie deactivation of CRISPR ribonucleases. Here is the latest research.

Cell-Type Specific Viral Vectors

Viral vectors are used in biological research and therapy to deliver genetic material into cells. However, the efficiency of viral vectors varies depending on the cell type. Here is the latest research on cell-type-specific viral vectors.