Next-Generation CRISPR Technologies and Their Applications in Gene and Cell Therapy

M. Alejandra Zeballos C., Thomas Gaj

Research output: Contribution to journalReview articlepeer-review


The emergence of clustered regularly interspaced short palindromic repeat (CRISPR) nucleases has transformed biotechnology by providing an easy, efficient, and versatile platform for editing DNA. However, traditional CRISPR-based technologies initiate editing by activating DNA double-strand break (DSB) repair pathways, which can cause adverse effects in cells and restrict certain therapeutic applications of the technology. To this end, several new CRISPR-based modalities have been developed that are capable of catalyzing editing without the requirement for a DSB. Here, we review three of these technologies: base editors, prime editors, and RNA-targeting CRISPR-associated protein (Cas)13 effectors. We discuss their strengths compared to traditional gene-modifying systems, we highlight their emerging therapeutic applications, and we examine challenges facing their safe and effective clinical implementation.

Original languageEnglish (US)
Pages (from-to)692-705
Number of pages14
JournalTrends in Biotechnology
Issue number7
StatePublished - Jul 2021


  • CRISPR-Cas13
  • base editing
  • gene therapy
  • prime editing

ASJC Scopus subject areas

  • Biotechnology
  • Bioengineering


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