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Updated: Aug 17, 2025

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
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Development of curative therapies for sickle cell disease

Yvette C Tanhehco1, Ghazala Nathu2, Ljiljana V Vasovic3

  • 1Department of Pathology and Cell Biology, Columbia University Irving Medical Center, New York, NY, United States.

Frontiers in Medicine
|December 12, 2022
PubMed

Insights

Recent Sickle Cell Disease (SCD) treatments, including new drugs and stem cell transplants, have improved patient outcomes. Gene therapy offers a promising potential cure, requiring expert multidisciplinary care for effective SCD management.

Area of Science:

  • Hematology
  • Genetics
  • Pharmacology

Background:

  • Sickle Cell Disease (SCD) management has advanced, improving survival and quality of life.
  • Current treatments include disease-modifying drugs and hematopoietic stem cell transplantation (HSCT).
  • HSCT offers a cure but is limited by donor availability.

Purpose of the Study:

  • To review recent advancements in SCD management.
  • To highlight emerging curative therapies like gene therapy.
  • To emphasize the need for multidisciplinary expertise in SCD care.

Main Methods:

  • Review of recent literature on SCD therapies.
  • Analysis of drug efficacy and transplantation outcomes.
  • Discussion of gene therapy approaches and clinical trial progress.

Main Results:

  • Disease-modifying drugs (e.g., hydroxyurea, L-glutamine, voxelotor, crizanlizumab) reduce SCD complications.
  • Expanded donor options for HSCT (cord blood, haploidentical) increase accessibility.
  • Gene therapy trials show promising efficacy for SCD cure.

Conclusions:

  • SCD management has significantly improved with new drug therapies and expanded transplantation options.
  • Gene therapy represents a highly promising curative approach for SCD.
  • Optimal SCD treatment necessitates a multidisciplinary expert team.

Keywords:
Sickle Cell Disease (SCD)disease modifying drugsgene therapyhematopoietic stem cell transplantation (HSCT)lentiviral vectors (LVS)

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