Gene Therapy for Neurofibromatosis Type 2-Related Schwannomatosis: Recent Progress, Challenges, and Future Directions.
Ruofei YuanBo WangYing WangPi-Nan LiuPublished in: Oncology and therapy (2024)
Neurofibromatosis type 2 (NF2)-related schwannomatosis is a rare autosomal dominant monogenic disorder caused by mutations in the NF2 gene. The hallmarks of NF2-related schwannomatosis are bilateral vestibular schwannomas (VS). The current treatment options for NF2-related schwannomatosis, such as observation with serial imaging, surgery, radiotherapy, and pharmacotherapies, have shown limited effectiveness and serious complications. Therefore, there is a critical demand for novel effective treatments. Gene therapy, which has made significant advancements in treating genetic diseases, holds promise for the treatment of this disease. This review covers the genetic pathogenesis of NF2-related schwannomatosis, the latest progress in gene therapy strategies, current challenges, and future directions of gene therapy for NF2-related schwannomatosis.
Keyphrases
- gene therapy
- signaling pathway
- lps induced
- oxidative stress
- pi k akt
- randomized controlled trial
- early stage
- minimally invasive
- high resolution
- immune response
- squamous cell carcinoma
- cell proliferation
- inflammatory response
- dna methylation
- big data
- drug induced
- percutaneous coronary intervention
- artificial intelligence
- radiation induced
- current status
- photodynamic therapy
- replacement therapy
- genome wide analysis