CRISPR Takes the Front Seat in CART-Cell Development

Claudia Manriquez-Roman, Elizabeth L. Siegler, Saad S. Kenderian

Research output: Contribution to journalArticlepeer-review


Chimeric antigen receptor T (CART)-cell immunotherapies have opened a door in the development of specialized gene therapies for hematological and solid cancers. Impressive response rates in pivotal trials led to the FDA approval of CART-cell therapy for certain hematological malignancies. However, autologous CART products are costly and time-intensive to manufacture, and most patients experience disease relapse within 1 year of CART administration. Additionally, CART-cell efficacy in solid tumors is extremely limited. CART-cell therapy is also associated with serious toxicities. Manufacturing difficulties, intrinsic T-cell defects, CART exhaustion, and treatment-associated toxicities are some of the current barriers to widespread adoption of CART-cell therapy. Genome editing tools such as CRISPR/Cas systems have demonstrated efficacy in further engineering CART cells to overcome these limitations. In this review, we will summarize the current approaches that use CRISPR to facilitate off-the-shelf CART products, increase CART-cell efficacy, and minimize CART-associated toxicities.

Original languageEnglish (US)
Pages (from-to)113-124
Number of pages12
Issue number2
StatePublished - Mar 2021

ASJC Scopus subject areas

  • Biotechnology
  • Pharmacology
  • Pharmacology (medical)


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