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November 11, 2025GenesOpen Access

Curing Sickle Cell Disease by Allogeneic Hematopoietic Stem Cell (HSC) Transplantation Toward In Vivo HSC Gene Therapy

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Authors

RKRina Kansal

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Overview

Review explores gene therapies for sickle cell disease, highlighting lentiviral-based approaches and genome editing.

Key Points

  • This review discusses recent advancements in gene therapies for sickle cell disease.
  • Reviewed two approved gene therapies for sickle cell disease: lentiviral-based and nonviral.
  • Explained mechanisms of action and differences between therapies.
  • Analyzed preclinical studies for in vivo HSC gene therapy and its potential.
  • Identified limitations of current therapies that require allogeneic HSCT.
  • Highlighted the need for gene therapies that eliminate HSCT requirements.
  • Discussed advancements in genome editing technologies for future treatments.

Cite This Study

Rina Kansal (2025) studied this question.

synapsesocial.com/papers/69252ea3c0ce034ddc3567f2https://doi.org/10.3390/genes16111367
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Also Consider

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

  1. 1Hematopoietic stem cell therapy with gene modification to treat sickle cell disease2025 · 4 citations
  2. 2The New Era of Curative Therapies for Sickle Cell Disease: A Comprehensive Review of Allogeneic Transplantation and Autologous Gene Therapy2026
  3. 3The Journey of Gene Therapy in Sickle Cell Disease: How Molecular Advances Meet Clinical Care2026
  4. 4Recent advancements in gene therapy for sickle cell disease and β-thalassemia2024 · 13 citations
  5. 5Current state of gene therapy in sickle cell disease2024 · 14 citations