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June 14, 2026EncyclopediaOpen Access

The New Era of Curative Therapies for Sickle Cell Disease: A Comprehensive Review of Allogeneic Transplantation and Autologous Gene Therapy

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Authors

AAAhmed AzeezTbilisi State Medical UniversityHVHarshitha VallabhaneniApollo Institute of Medical Sciences & ResearchATAdhith TheyverAdvanced Emissions Solutions (United States)

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Overview

Comprehensive review evaluates allogeneic transplantation and autologous gene therapy outcomes in sickle cell disease, suggesting advances in access and treatment efficacy.

Key Points

  • This review aims to synthesize the mechanisms of sickle cell disease and evaluate curative therapies, focusing on allogeneic and autologous approaches.
  • Evaluated clinical outcomes of allogeneic hematopoietic stem cell transplantation using various donor sources.
  • Analyzed autologous gene therapies including lentiviral gene addition and CRISPR-Cas9 editing.
  • Discussed clinical decision-making paradigms and future innovations for global access.
  • Allogeneic HSCT offers high survival rates but is limited by donor availability and risk of GVHD.
  • Autologous gene therapies like Lyfgenia and Casgevy provide innovative, donor-independent options with no GVHD risk.
  • Emerging therapies may improve access and outcomes for patients with sickle cell disease.

Cite This Study

Azeez et al. (2026) studied this question.

synapsesocial.com/papers/6a2e4608b1cc60ccdea8aec0https://doi.org/10.3390/encyclopedia6060131
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Also Consider

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

  1. 1The Journey of Gene Therapy in Sickle Cell Disease: How Molecular Advances Meet Clinical Care2026
  2. 2Curing Sickle Cell Disease by Allogeneic Hematopoietic Stem Cell (HSC) Transplantation Toward In Vivo HSC Gene Therapy2025
  3. 3Sickle cell disease: understanding pathophysiology, clinical features and advances in gene therapy approaches2025 · 16 citations
  4. 4Current state of gene therapy in sickle cell disease2024 · 14 citations
  5. 5Hematopoietic stem cell therapy with gene modification to treat sickle cell disease2025 · 4 citations