Synapse
⌘+K
Synapse
PulseExploreClubsResearchersJournals
Instagram
HomeClubsExplore
May 27, 2026CellsOpen Access

The Journey of Gene Therapy in Sickle Cell Disease: How Molecular Advances Meet Clinical Care

View Full Paper
Ask AI
Bookmark
Share

Authors

MTMagalie TardifCentre Hospitalier Universitaire Sainte-JustineMSManon SabyCentre Hospitalier Universitaire Sainte-JustineSFStéphanie FortéHEC Montréal

Discussion

Loading...

Member takes

Implication

Review examines gene therapy's role in modifying sickle cell disease, highlighting clinical benefits and limitations.

Key Points

  • The review explores the evolution of gene therapy for sickle cell disease and its clinical applications.
  • Analyzed two approved autologous gene therapies, exa-cel and lovo-cel, and their mechanisms of action.
  • Discussed engineering methods, including lentiviral and CRISPR-based platforms for gene therapy.
  • Evaluated the clinical evidence on disease modification and assessed barriers to access.
  • Autologous gene therapies demonstrated durable disease modification with acceptable short-term toxicity.
  • Gene therapy is positioned as a viable curative option compared to traditional treatments, despite access barriers.
  • Identified critical research priorities including long-term safety, pediatric trials, and effectiveness studies.

Cite This Study

Tardif et al. (2026) studied this question.

synapsesocial.com/papers/6a168ae40c924ddd1bd59aa5https://doi.org/10.3390/cells15100939
View Full Paper
Ask AI
Bookmark
Share

Also Consider

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

  1. 1Lovotibeglogene Autotemcel Gene Therapy for Sickle Cell Disease: 60 Months Follow-up2024 · 3 citations
  2. 2Navigating equity in global access to genome therapy expanding access to potentially transformative therapies and benefiting those in need requires global policy changes2024 · 19 citations
  3. 3Human beta-globin messenger RNA. III. Nucleotide sequences derived from complementary DNA.1977 · 228 citations
  4. 4Gene Therapy for β-Hemoglobinopathies2017 · 127 citations