Literature DB >> 35798006

Therapeutic in vivo delivery of gene editing agents.

Aditya Raguram1, Samagya Banskota1, David R Liu2.   

Abstract

In vivo gene editing therapies offer the potential to treat the root causes of many genetic diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to safely and efficiently deliver gene editing agents to relevant organs and tissues in vivo. Here, we review current delivery technologies that have been used to enable therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like particles. Since no single delivery modality is likely to be appropriate for every possible application, we compare the benefits and drawbacks of each method and highlight opportunities for future improvements.
Copyright © 2022. Published by Elsevier Inc.

Entities:  

Mesh:

Substances:

Year:  2022        PMID: 35798006      PMCID: PMC9454337          DOI: 10.1016/j.cell.2022.03.045

Source DB:  PubMed          Journal:  Cell        ISSN: 0092-8674            Impact factor:   66.850


  219 in total

Review 1.  Gene therapy for neurological disorders: progress and prospects.

Authors:  Benjamin E Deverman; Bernard M Ravina; Krystof S Bankiewicz; Steven M Paul; Dinah W Y Sah
Journal:  Nat Rev Drug Discov       Date:  2018-08-10       Impact factor: 84.694

Review 2.  The mechanism of double-strand DNA break repair by the nonhomologous DNA end-joining pathway.

Authors:  Michael R Lieber
Journal:  Annu Rev Biochem       Date:  2010       Impact factor: 23.643

3.  Adeno-associated virus (AAV) vectors achieve prolonged transgene expression in mouse myocardium and arteries in vivo: a comparative study with adenovirus vectors.

Authors:  Giuseppe Vassalli; Hansruedi Büeler; Jean Dudler; Ludwig K von Segesser; Lukas Kappenberger
Journal:  Int J Cardiol       Date:  2003-08       Impact factor: 4.164

4.  Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10.

Authors:  Morgan L Maeder; Michael Stefanidakis; Christopher J Wilson; Reshica Baral; Luis Alberto Barrera; George S Bounoutas; David Bumcrot; Hoson Chao; Dawn M Ciulla; Jennifer A DaSilva; Abhishek Dass; Vidya Dhanapal; Tim J Fennell; Ari E Friedland; Georgia Giannoukos; Sebastian W Gloskowski; Alexandra Glucksmann; Gregory M Gotta; Hariharan Jayaram; Scott J Haskett; Bei Hopkins; Joy E Horng; Shivangi Joshi; Eugenio Marco; Rina Mepani; Deepak Reyon; Terence Ta; Diana G Tabbaa; Steven J Samuelsson; Shen Shen; Maxwell N Skor; Pam Stetkiewicz; Tongyao Wang; Clifford Yudkoff; Vic E Myer; Charles F Albright; Haiyan Jiang
Journal:  Nat Med       Date:  2019-01-21       Impact factor: 53.440

5.  Engraftment of a galactose receptor footprint onto adeno-associated viral capsids improves transduction efficiency.

Authors:  Shen Shen; Eric D Horowitz; Andrew N Troupes; Sarah M Brown; Nagesh Pulicherla; Richard J Samulski; Mavis Agbandje-McKenna; Aravind Asokan
Journal:  J Biol Chem       Date:  2013-08-12       Impact factor: 5.157

6.  Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo.

Authors:  John A Zuris; David B Thompson; Yilai Shu; John P Guilinger; Jeffrey L Bessen; Johnny H Hu; Morgan L Maeder; J Keith Joung; Zheng-Yi Chen; David R Liu
Journal:  Nat Biotechnol       Date:  2014-10-30       Impact factor: 54.908

7.  In vivo base editing of post-mitotic sensory cells.

Authors:  Wei-Hsi Yeh; Hao Chiang; Holly A Rees; Albert S B Edge; David R Liu
Journal:  Nat Commun       Date:  2018-06-05       Impact factor: 14.919

8.  Transient Retrovirus-Based CRISPR/Cas9 All-in-One Particles for Efficient, Targeted Gene Knockout.

Authors:  Yvonne Knopp; Franziska K Geis; Dirk Heckl; Stefan Horn; Thomas Neumann; Johannes Kuehle; Janine Meyer; Boris Fehse; Christopher Baum; Michael Morgan; Johann Meyer; Axel Schambach; Melanie Galla
Journal:  Mol Ther Nucleic Acids       Date:  2018-09-13       Impact factor: 8.886

Review 9.  Engineering precision nanoparticles for drug delivery.

Authors:  Michael J Mitchell; Margaret M Billingsley; Rebecca M Haley; Marissa E Wechsler; Nicholas A Peppas; Robert Langer
Journal:  Nat Rev Drug Discov       Date:  2020-12-04       Impact factor: 84.694

10.  The impact of lentiviral vector genome size and producer cell genomic to gag-pol mRNA ratios on packaging efficiency and titre.

Authors:  Nathan P Sweeney; Conrad A Vink
Journal:  Mol Ther Methods Clin Dev       Date:  2021-04-16       Impact factor: 6.698

View more
  2 in total

1.  Precision genome editing in the eye.

Authors:  Susie Suh; Elliot H Choi; Aditya Raguram; David R Liu; Krzysztof Palczewski
Journal:  Proc Natl Acad Sci U S A       Date:  2022-09-19       Impact factor: 12.779

Review 2.  Biocompatible Iron Oxide Nanoparticles for Targeted Cancer Gene Therapy: A Review.

Authors:  Jinsong Zhang; Tianyuan Zhang; Jianqing Gao
Journal:  Nanomaterials (Basel)       Date:  2022-09-24       Impact factor: 5.719

  2 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.