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Updated: Dec 11, 2025
Genome Editing in Mammalian Cell Lines using CRISPR-Cas
Published on: April 11, 2019
Translating CRISPR-Cas Therapeutics: Approaches and Challenges
Lavina Sierra Tay1, Nathan Palmer2, Rebecca Panwala3
1Laboratory of Synthetic Biology and Genome Editing Therapeutics, Genome Institute of Singapore, Agency for Science, Technology and Research, Singapore, Singapore.; University of California San Diego, La Jolla, California, USA.
Abstract:
CRISPR-Cas clinical trials have begun, offering a first glimpse at how DNA and RNA targeting could enable therapies for many genetic and epigenetic human diseases. The speedy progress of CRISPR-Cas from discovery and adoption to clinical use is built on decades of traditional gene therapy research and belies the multiple challenges that could derail the successful translation of these new modalities. Here, we review how CRISPR-Cas therapeutics are translated from technological systems to therapeutic modalities, paying particular attention to the therapeutic cascade from cargo to delivery vector, manufacturing, administration, pipelines, safety, and therapeutic target profiles. We also explore potential solutions to some of the obstacles facing successful CRISPR-Cas translation. We hope to illuminate how CRISPR-Cas is brought from the academic bench toward use in the clinic.
Insights
CRISPR-Cas gene editing therapies are advancing rapidly, with clinical trials underway for genetic diseases. This review examines the translation of CRISPR-Cas technology into clinical applications, addressing key challenges and potential solutions for successful therapeutic development.
Area of Science:
- Biotechnology and Genetic Medicine
- Molecular Biology and Genomics
Background:
- CRISPR-Cas technology represents a significant advancement in gene editing.
- Early clinical trials are exploring its potential for treating genetic and epigenetic diseases.
- Translating CRISPR-Cas from the lab to the clinic faces numerous hurdles.
Purpose of the Study:
- To review the process of translating CRISPR-Cas therapeutics from technological systems to clinical modalities.
- To identify and analyze challenges in the therapeutic cascade, including delivery, manufacturing, and safety.
- To explore potential solutions for overcoming obstacles in CRISPR-Cas clinical translation.
Main Methods:
- Review of current CRISPR-Cas research and clinical trial data.
- Analysis of the therapeutic cascade for CRISPR-Cas based medicines.
- Identification of challenges and proposed solutions in CRISPR-Cas development.
Main Results:
- CRISPR-Cas clinical trials are initiating, demonstrating early therapeutic promise.
- Successful translation requires addressing complex issues in cargo, delivery, manufacturing, and administration.
- Safety and therapeutic target profiling are critical for clinical success.
Conclusions:
- The rapid advancement of CRISPR-Cas technology is built upon foundational gene therapy research.
- Overcoming translational challenges is crucial for realizing the full therapeutic potential of CRISPR-Cas.
- This review provides insights into the pathway of CRISPR-Cas from research to clinical application.
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