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Updated: Oct 17, 2025
07:56
Genome Editing in Mammalian Cell Lines using CRISPR-Cas
Published on: April 11, 2019
22.3K
CRISPR-derived genome editing therapies: Progress from bench to bedside.
Holly A Rees1, Alex C Minella1, Cameron A Burnett2
1Beam Therapeutics, Cambridge, MA, USA.
Summary
CRISPR genome editing offers precise DNA manipulation. This review covers its development, cellular mechanisms, therapeutic considerations like off-target edits, and challenges in clinical application for novel therapies.
Area of Science:
- Molecular Biology
- Genetics
- Biotechnology
Background:
- CRISPR-derived technologies allow precise DNA sequence editing in the human genome.
- Understanding CRISPR nucleases and DNA base editors is crucial for advancing gene editing applications.
Purpose of the Study:
- To review the development and cellular mechanisms of CRISPR nucleases and DNA base editors.
- To discuss critical factors for translating genome editing tools into therapeutic agents.
- To identify challenges in the clinical application of CRISPR-based therapies.
Main Methods:
- Literature review of CRISPR-derived genome editing technologies.
- Analysis of cellular mechanisms of CRISPR nucleases and DNA base editors.
- Examination of factors influencing therapeutic development and clinical translation.
Main Results:
- CRISPR technologies enable precise DNA manipulation.
- Key considerations for therapeutic development include off-target edits and characterization methods.
- Clinical translation faces challenges in manufacturing, regulation, and trial design.
Conclusions:
- CRISPR genome editing holds significant therapeutic potential.
- Addressing off-target effects and manufacturing/regulatory hurdles is essential for clinical success.
- Further research and development are needed to optimize CRISPR-based therapies for patient use.
Keywords:
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