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March 1, 2024Molecular Therapy — Methods & Clinical DevelopmentOpen Access

Optimizing lentiviral genomic integrations to cure beta-thalassemia: The least required for success?

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SRStefano RivellaUniversity City Science Center

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Stefano Rivella (2024) studied this question.

synapsesocial.com/papers/68e7681cb6db6435876dd446https://doi.org/10.1016/j.omtm.2024.101222
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Also Consider

Synapse has enriched 4 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Correction of Sickle Cell Disease in Transgenic Mouse Models by Gene Therapy2001 · 587 citations
  2. 2Drug product attributes predict clinical efficacy in betibeglogene autotemcel gene therapy for β-thalassemia2023 · 8 citations
  3. 3Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease2021 · 432 citations
  4. 4Gene Therapy in a Patient with Sickle Cell Disease2017 · 695 citations