Gene therapy

Where the papers sit

13 papers from the last 6 months study gene therapy directly · updated from PubMed 21 Oct 2026

• Rare Disease Gene Delivery

  AAV delivery is being adapted for both systemic and tissue-specific treatment of rare disease. Intravenous vectors improve survival or phenotype in Sandhoff disease and Cockayne syndrome models, while liver targeting addresses progressive familial intrahepatic cholestasis. CNS access remains a central challenge, prompting focused ultrasound at the blood-brain barrier and inhibitory-neuron targeting with a compact GAD67 promoter. Clinical translation also depends on OTOF variant classification and safer nonviral nanocarriers for ocular delivery.

  7 papers · 53.8%

• Sickle Cell Treatment Comparisons

  Gene therapy and allogeneic hematopoietic transplantation are being compared with standard care for sickle-cell disease. Evaluation extends beyond clinical outcomes to blood rheology after treatment, including the effects of hydroxycarbamide and stem-cell transplantation. Cost-effectiveness analysis is becoming part of decisions about haploidentical transplant and gene therapy. Regulatory frameworks remain an external constraint on translating these options into routine care.

  3 papers · 23.1%

• Triple-Negative Breast Cancer Combinations

  Triple-negative breast cancer treatment is moving toward multimodal combinations that pair targeted delivery with immune, apoptotic, or ferroptotic mechanisms. Antibody-drug conjugates are combined with cmLumiOpto, while siATAD3A carriers use sonodynamic activation to promote co-gene-ferroptosis therapy. A separate strategy enhances PD-1-mediated immunity through Lactobacillus rhamnosus, gut microbiota, and tyrosine metabolism. The evidence remains preclinical, leaving durability, toxicity, and comparative benefit unresolved.

  3 papers · 23.1%