Literature DB >> 31728915

Gene Editing in Human Pluripotent Stem Cells: Recent Advances for Clinical Therapies.

Hatice Burcu Şişli1, Taha Bartu Hayal1, Selin Seçkin1, Selinay Şenkal1, Binnur Kıratlı1, Fikrettin Şahin1, Ayşegül Doğan2.   

Abstract

The identification of human embryonic stem cells and reprogramming technology to obtain induced pluripotent stem cells from adult somatic cells have provided unique opportunity to create human disease models, gene editing strategies and cell therapy options.Development of pluripotent stem cells from somatic cells and genomic manipulation tools enabled to use site specific nucleases in the cell therapy research. Identification of efficient gene manipulation, safe differentiation and use will provide a novel strategy to treat many diseases in the near future. Current available registered clinical trials clearly indicate the need for pluripotent stem cell and gene therapy treatment options. Although gene editing based pluripotent stem cell research is a popular field for research worldwide, improvement of clinical approaches for treatment still remains to be investigated. In this review, we summarized the current situation of gene editing based pluripotent cell therapy developments and applications in clinics.

Entities:  

Keywords:  Cell therapy; Clinical trial; Gene therapy; Pluripotent stem cell; Regenerative medicine

Mesh:

Year:  2020        PMID: 31728915     DOI: 10.1007/5584_2019_439

Source DB:  PubMed          Journal:  Adv Exp Med Biol        ISSN: 0065-2598            Impact factor:   2.622


  64 in total

Review 1.  Genome editing: a robust technology for human stem cells.

Authors:  Arun Pandian Chandrasekaran; Minjung Song; Suresh Ramakrishna
Journal:  Cell Mol Life Sci       Date:  2017-04-12       Impact factor: 9.261

Review 2.  Xanthomonas AvrBs3 family-type III effectors: discovery and function.

Authors:  Jens Boch; Ulla Bonas
Journal:  Annu Rev Phytopathol       Date:  2010       Impact factor: 13.078

3.  TALEs of genome targeting.

Authors:  Jens Boch
Journal:  Nat Biotechnol       Date:  2011-02       Impact factor: 54.908

Review 4.  Genome engineering with zinc-finger nucleases.

Authors:  Dana Carroll
Journal:  Genetics       Date:  2011-08       Impact factor: 4.562

Review 5.  A decade of discovery: CRISPR functions and applications.

Authors:  Rodolphe Barrangou; Philippe Horvath
Journal:  Nat Microbiol       Date:  2017-06-05       Impact factor: 17.745

6.  Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting.

Authors:  Chia-Wei Chang; Yi-Shin Lai; Erik Westin; Alireza Khodadadi-Jamayran; Kevin M Pawlik; Lawrence S Lamb; Frederick D Goldman; Tim M Townes
Journal:  Cell Rep       Date:  2015-08-28       Impact factor: 9.423

7.  Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.

Authors:  Niclas E Bengtsson; John K Hall; Guy L Odom; Michael P Phelps; Colin R Andrus; R David Hawkins; Stephen D Hauschka; Joel R Chamberlain; Jeffrey S Chamberlain
Journal:  Nat Commun       Date:  2017-02-14       Impact factor: 14.919

8.  Combining Induced Pluripotent Stem Cells and Genome Editing Technologies for Clinical Applications.

Authors:  Chia-Yu Chang; Hsiao-Chien Ting; Hong-Lin Su; Jing-Ren Jeng
Journal:  Cell Transplant       Date:  2018-05-28       Impact factor: 4.064

9.  Using Zinc Finger Nuclease Technology to Generate CRX-Reporter Human Embryonic Stem Cells as a Tool to Identify and Study the Emergence of Photoreceptors Precursors During Pluripotent Stem Cell Differentiation.

Authors:  Joseph Collin; Carla B Mellough; Birthe Dorgau; Stefan Przyborski; Inmaculada Moreno-Gimeno; Majlinda Lako
Journal:  Stem Cells       Date:  2015-11-26       Impact factor: 6.277

Review 10.  CRISPR-Cas immunity, DNA repair and genome stability.

Authors:  Andrew Cubbon; Ivana Ivancic-Bace; Edward L Bolt
Journal:  Biosci Rep       Date:  2018-09-20       Impact factor: 3.840

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  1 in total

Review 1.  Transgene Delivery to Human Induced Pluripotent Stem Cells Using Nanoparticles.

Authors:  Megan A Yamoah; Phung N Thai; Xiao-Dong Zhang
Journal:  Pharmaceuticals (Basel)       Date:  2021-04-06
  1 in total

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