Skip to content

#genetic therapy

2 public questions tagged with this topic.

Cancer gene therapy often uses strategy of:

Cancer gene therapy predominantly adopts tumoricidal strategy rather than restoration tumor suppressor because cancer genome carries multiple gain-of-function oncogenes KRAS MYC aneuploidy heterogeneity complicating simple addition. Methods eliminate malignant clone through introduction genes triggering death or immune recruitment. Oncolytic adenoviruses engineered deletion E1B 55 kDa protein normally inactivating p53 replicate selectively p53-deficient cancers lysing cells via viral burst releasing PAMPs tumor antigens danger signals ATP calreticulin HMGB1 driving immunogenic cell death. Suic

Ref: Nature Reviews Cancer Gene Therapy Oncolytic Mechanisms 2021; NCI Cancer Gene Therapy Targeted Killing; Molecular Therapy Oncolytic Viruses 2021.

Somatic gene therapy differs from germline gene therapy because somatic therapy:

Somatic gene therapy restricts genetic modification to differentiated somatic lineages such as hematopoietic stem cells, hepatocytes, myofibers, retinal pigment epithelium, or T lymphocytes, preserving integrity of primordial germ cells in gonadal ridges. Recombinant lentiviral vectors pseudotyped with VSV-G bind LDL receptor, entering CD34+ stem cells after cytokine stimulation with SCF, TPO, FLT3L that drives cell cycling required for reverse transcription and integration. Integrated provirus flanked by LTRs replicates synchronously with host chromosomes during mitosis, ensuring inheritance

Ref: CCMO Netherlands Somatic vs Germline Therapy; MedlinePlus Somatic Gene Editing; Watson Mol Biol Gene Chap 12.