Sickle cell disease-specific barriers to in vivo hematopoietic stem cell gene therapy
Summary
Gene therapy for individuals living with sickle cell disease is a transformative therapy, ameliorating many of its severe symptoms. Nevertheless, ex vivo administration of gene therapy coupled with sickle cell disease specific obstacles, complicate and limit widespread adoption. In this review, we discuss current strategies to develop in vivo gene delivery technologies, highlighting different approaches and their potential benefits and limitations in the context of sickle cell disease.
Gene therapy for individuals living with sickle cell disease is a transformative therapy, ameliorating many of its severe symptoms. Nevertheless, ex vivo administration of gene therapy coupled with sickle cell disease specific obstacles, complicate and limit widespread adoption. In this review, we discuss current strategies to develop in vivo gene delivery technologies, highlighting different approaches and their potential benefits and limitations in the context of sickle cell disease. Additionally, we examine editing challenges and outline how in vivo gene editing may be evaluated for therapeutic benefit.
Abstract from PubMed (PMID 42957504). This entry was added automatically by our daily literature monitor because it matches the topics we follow; read the full paper at the original source.
Related articles
Mathematical modeling in mRNA therapeutics: towards a pathway to individualized treatment protocols
Molecular pharmaceuticsCombined siRNA Targeting IκBα and Stat6 Promotes Macrophage Reprogramming toward Cancer Immunotherapy
The Journal of clinical investigationA single liver-sparing myotropic AAV ameliorates muscular dystrophy in humanized mice by base editing
Let's engineer the next delivery breakthrough together
We co-develop nanocarrier and biosensing programmes with pharma, biotech and academic groups — from target selection through scale-up.