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September 10, 2025

CRISPR-Cas9 Mediated Gene Editing in Inherited Hematologic Disorders

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Authors

SCSrikumar ChakravarthiSEGi UniversityJJJohn Paul JudsonUniversiti Tunku Abdul RahmanBKBarani KarikalanMahsa University

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Overview

Pre-clinical testing reports improved blood function in animal models with CRISPR-Cas9, suggesting potential for treatment.

Key Points

  • CRISPR-Cas9 shows promising efficiency in editing mutations causing hematologic disorders, with improvements observed.
  • Edit efficiencies ranged between 42% and 65% in animal models, leading to better hemoglobin levels and clotting factors.
  • Pre-clinical testing in mice, dogs, and primates supports the safety and effectiveness of CRISPR-Cas9 therapies.
  • The research highlights the importance of animal models in bridging laboratory discoveries and potential human treatments.

Cite This Study

Chakravarthi et al. (2025) studied this question.

synapsesocial.com/papers/68c1ad4f54b1d3bfb60e4fa9https://doi.org/10.64062/jpgmb.vol1.issue4.2
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1CRISPR-Cas9-Mediated Gene Editing in Hematological Disorders: Advancing Translational and Clinical Applications2026
  2. 2The CRISPR-Cas9 Technology Used for the Treatment of Single-Gene Genetic Diseases2025
  3. 3CRISPR application in hematological disorders: from bench to bedside2026
  4. 4THERAPEUTIC CRISPR/CAS9 GENOME EDITING TOOL FOR TREATING SICKLE CELL DISEASE2024
  5. 5CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments2024 · 104 citations