The Journey of Gene Therapy in Sickle Cell Disease: How Molecular Advances Meet Clinical Care.
Level 5 - mechanism / opinion, no new human data
Narrative review with expert commentary and no systematic review methodology
PubMed 42193948 · doi:10.3390/cells15100939
What was done
This narrative review summarizes molecular mechanisms, engineering strategies (lentiviral and CRISPR platforms), and clinical evidence for approved autologous gene therapies (exagamglogene autotemcel and lovotibeglogene autotemcel) in sickle cell disease. It evaluates clinical positioning against allogeneic transplantation and standard treatments, alongside financial, ethical, and psychosocial access barriers.
What was found
The abstract reports no numerical findings, effect sizes, or quantitative safety/efficacy statistics. It qualitatively reports that accumulated clinical evidence demonstrates durable disease modification with acceptable short-term toxicity, and identifies research priorities including long-term safety, pediatric trials, and non-genotoxic conditioning.
Why it matters
It synthesizes the clinical transition of newly approved curative gene therapies for sickle cell disease and frames key logistical and therapeutic barriers to broad adoption.
Limits
This is a narrative review presenting no new empirical data or systematic meta-analysis. The abstract provides no specific quantitative metrics or patient cohort sizes.
Cited by
- supports Most regulatory approved gene therapies work by gene addition rather than by gene editing.