CRISPR application in hematological disorders: from bench to bedside.

Area of Science:

• Hematology
• Genetics
• Immunology

Background:

• CRISPR-Cas genome editing has transitioned from research to clinical application in hematology.
• Successful treatments for inherited blood disorders demonstrate the safety and efficacy of genetic modification in hematopoietic cells.

Purpose of the Study:

• To review recent CRISPR-based advancements in benign and malignant hematologic diseases.
• To compare different CRISPR editing modalities and discuss clinical trial data.
• To identify translational challenges and future priorities for CRISPR therapeutics in hematology.

Main Methods:

• Review of recent CRISPR-based advances across benign and malignant hematologic diseases.
• Comparison of major editing modalities: nuclease-mediated disruption, base editing, prime editing, and CRISPR-based transcriptional modulation.
• Synthesis of preclinical studies and emerging clinical trial data.

Main Results:

• CRISPR editing is effective for inherited blood diseases and shows promise in understanding and treating hematologic malignancies.
• Engineered cellular therapies using CRISPR are being developed for enhanced antitumor activity.
• Various CRISPR editing modalities offer different approaches for genetic modification.

Conclusions:

• CRISPR technology is a powerful tool in hematology, with significant progress in treating genetic blood disorders and cancer.
• Addressing challenges in delivery, scalability, safety, and long-term effects is crucial for broader clinical adoption.
• Continued innovation in CRISPR technologies will drive precise, durable, and mechanism-informed therapeutic strategies in hematology.