Activating human genes with zinc finger proteins, transcription activator-like effectors and CRISPR/Cas9 for gene therapy and regenerative medicine

Charles A. Gersbach, Pablo Perez-Pinera

Research output: Contribution to journalReview article

Abstract

New technologies have recently been developed to control the expression of human genes in their native genomic context by engineering synthetic transcription factors that can be targeted to any DNA sequence. The ability to precisely regulate any gene as it occurs naturally in the genome provides a means to address a variety of diseases and disorders. This approach also circumvents some of the traditional challenges of gene therapy. In this editorial, we review the technologies that have enabled targeted human gene activation, including the engineering of transcription factors based on zinc finger proteins, transcription activator-like effectors and the CRISPR/Cas9 system. Additionally, we highlight examples in which these methods have been developed for therapeutic applications and discuss challenges and opportunities.

Original languageEnglish (US)
Pages (from-to)835-839
Number of pages5
JournalExpert Opinion on Therapeutic Targets
Volume18
Issue number8
DOIs
StatePublished - Aug 2014
Externally publishedYes

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Clustered Regularly Interspaced Short Palindromic Repeats
Gene therapy
Regenerative Medicine
Zinc Fingers
Transcription
Genetic Therapy
Zinc
Transcription Factors
Genes
Technology
Transcriptional Activation
Proteins
Genome
Gene Expression
DNA sequences
Chemical activation
Transcription Activator-Like Effectors
Therapeutics

Keywords

  • Cas9
  • CRISPR
  • Gene editing
  • Gene regulation
  • Gene therapy
  • Genetic reprogramming
  • Protein engineering
  • Transcription activator-like effector
  • Transcription factor
  • Zinc finger

ASJC Scopus subject areas

  • Drug Discovery
  • Pharmacology
  • Clinical Biochemistry
  • Molecular Medicine

Cite this

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