gene therapy

Articles

• AAV vectorsGeneticsEngineered adeno-associated viruses used to carry therapeutic genes into human cells, the workhorse of in vivo gene therapy and the source of most of its limits.TRL 9presentEstablished
• Access and inequalitySocietyThe problem of who can obtain expensive biomedical interventions, and whether biotechnology will convert economic inequality into biological inequality.presentEstablished
• Base editingGeneticsA genome editing method that chemically converts one DNA base into another at a targeted site without cutting both strands of the double helix.TRL 7late 2020sEmerging
• CasgevyGeneticsThe first approved CRISPR-based medicine, which disrupts a fetal-haemoglobin repressor in a patient's own blood stem cells to treat sickle cell disease and beta thalassaemia.TRL 9presentEmerging
• CRISPR–Cas9GeneticsA bacterial defence system repurposed as a programmable DNA-cutting tool, in which a short guide RNA directs the Cas9 enzyme to a chosen sequence in almost any genome.TRL 9presentEstablished
• Epigenetic reprogrammingLongevityThe use of pluripotency factors to reset age-associated marks on DNA and chromatin, restoring youthful gene expression in cells without converting them into stem cells.TRL 32030sExperimental
• Epigenome editingGeneticsTargeted modification of the chemical marks that control gene expression, switching a gene off or on without altering the underlying DNA sequence.TRL 62030sExperimental
• Gene dopingEnhancementThe non-therapeutic use of gene transfer or gene regulation to improve athletic performance, prohibited in sport since the early 2000s with no publicly confirmed case.late 2020sExperimental
• Gene therapy for agingLongevityThe use of delivered genes to modify aging biology systemically, demonstrated in mice with telomerase and other constructs and untested in any controlled human trial.TRL 32040sExperimental
• Lipid nanoparticlesGeneticsFour-component lipid vesicles that carry mRNA and other nucleic acids into cells, the delivery system behind mRNA vaccines and the first systemic in vivo CRISPR therapies.TRL 9presentEstablished
• Myostatin inhibitionEnhancementBlockade of the growth factor myostatin to increase skeletal muscle mass, an effect reproduced across species that has repeatedly failed to translate into functional benefit in humans.TRL 7late 2020sExperimental
• Off-target effects in genome editingGeneticsUnintended DNA changes caused by genome editors, either at sites resembling the target or at the target itself, and the assays used to find them.presentEstablished
• Partial reprogrammingLongevityTransient expression of pluripotency factors that resets age-associated epigenetic marks in a cell while stopping short of erasing its differentiated identity.TRL 32030sExperimental
• Prime editingGeneticsA genome editing method in which a nicking Cas9 fused to a reverse transcriptase writes a new DNA sequence specified by an extended guide RNA.TRL 62030sExperimental
• Somatic gene therapyGeneticsGenetic modification of a living person's body cells to treat disease, producing a change that is not passed on to their children.TRL 9presentEstablished