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The 66 pages that link to Gene therapy, each with the reason it gives.

• Horizontal gene transferRelated: Viral vectors deliver therapeutic genes by a designed transfer between cells.

• Gene expressionRelated: Therapeutic designs can change expression of a target gene or supply a functional product.

• Molecular biologyRelated: It turns knowledge of genes and gene expression into therapeutic intervention.

• GeneRelated: Some therapies add, replace, or modify genes to change cell function.

• Hematopoietic stem cell transplantationCompared with: Some inherited blood disorders can be treated by modifying a patient's cells rather than using donor cells.

• PlasmidRelated: Plasmids can deliver therapeutic genes in some experimental and clinical approaches.

• RNA interferenceCompared with: Unlike many gene therapies, RNAi drugs usually suppress expression without replacing a defective gene.

• VirusRelated: Viral vectors are tools for delivering therapeutic genes.

• Recombinant DNARelated: Some gene therapies use engineered DNA constructs to supply or regulate genetic instructions.

• BiotechnologyBroader topic: It uses biological delivery systems to alter or supplement genes for therapeutic effect.

• CRISPR-Cas9Related: Cas9 can be used to alter disease-associated sequences in therapeutic settings.

• Genetic engineeringRelated: Genetic engineering supplies methods for changing genes in therapeutic cells.

• Gene regulatory networkRelated: Therapies can target regulatory genes or alter expression in specific cells.

• Regenerative medicineRelated: It can support regeneration by changing cell behavior, but genetic modification alone is not tissue regeneration.

• Enzyme replacement therapyCompared with: It aims to make cells produce a therapeutic protein rather than repeatedly infusing it.

• CellRelated: Gene therapy targets cells to alter the instructions driving a disease.

• Duchenne muscular dystrophyRelated: Micro-dystrophin gene therapies aim to provide muscle cells with instructions for a shortened dystrophin protein.

• ImmunodeficiencyRelated: For selected inherited deficiencies, corrected stem cells can rebuild functional immune lineages.

• PhenylketonuriaCompared with: Unlike established dietary and drug treatments, it is not routine standard care for phenylketonuria.

• Severe combined immunodeficiencyRelated: For selected SCID types, corrected patient stem cells can restore production of the missing protein.

• Genetic disorderRelated: Some genetic disorders can be treated by replacing, editing, or regulating genetic material.

• Genome editingNarrower topic: Genome editing is one strategy within the broader field of gene therapy.

• Paul BergRelated: Gene therapy illustrates a later medical direction made possible by recombinant DNA techniques.

• Molecular geneticsRelated: Understanding genes at the molecular level enabled attempts to correct genetic disease.

• Biologic therapyCompared with: Unlike most biologics, gene therapy aims to alter cells’ genetic instructions.

• LeukodystrophyRelated: Gene therapies have become treatment options for selected leukodystrophies, including metachromatic leukodystrophy.

• Lipid nanoparticleNarrower topic: Lipid nanoparticles are one nonviral platform for delivering genetic medicines.

• AchromatopsiaRelated: Clinical research investigates gene replacement for specific inherited forms.

• Cell therapyCompared with: It can overlap with cell therapy, but its defining intervention is genetic rather than cellular transfer.

• Angelman syndromeRelated: UBE3A delivery is being studied as a way to restore gene function in affected neurons.

• Antisense oligonucleotideCompared with: Gene therapies often seek durable genetic change, while antisense effects usually depend on continued drug exposure.

• BiopharmaceuticalBroader topic: It is a biopharmaceutical approach that acts through genetic material rather than a conventional active ingredient.

• Leber's hereditary optic neuropathyNarrower topic: Clinical research has tested gene therapy approaches to address LHON-associated mitochondrial mutations.

• Zinc finger nucleaseRelated: Zinc finger nucleases have been developed to modify disease-related genes in cells.

• Chronic granulomatous diseaseRelated: Gene addition is being developed to restore NADPH oxidase function in blood-forming cells.

• Emmanuelle CharpentierRelated: CRISPR-Cas9 has enabled experimental approaches to correcting disease-causing mutations.

• Human enhancementRelated: Gene-editing techniques raise the possibility of changing traits beyond disease treatment.

• OsteopetrosisRelated: Gene-based correction could target the inherited defect, but safe durable delivery remains challenging.

• TALENRelated: TALEN editing has been investigated as a way to modify therapeutic cells ex vivo.

• CiliopathyCompared with: It offers a possible route to treating inherited ciliary defects, but delivery across affected tissues is challenging.

• Primary immunodeficiencyRelated: For selected inherited immunodeficiencies, corrected patient cells can restore a missing gene function.

• Rett syndromeRelated: MECP2 replacement is under study, but precise dosage control is a major challenge.

• TransgenesisCompared with: It also delivers genetic material, but aims to treat patients rather than create transgenic organisms.

• Alpha-1 antitrypsin deficiencyRelated: Researchers are investigating ways to restore alpha-1 antitrypsin production.

• HematologyRelated: It can address the inherited cause of some blood disorders, but durability and access remain concerns.

• Leber congenital amaurosisNarrower topic: The RPE65 treatment established a gene-replacement approach for one molecular subtype.

• AdrenoleukodystrophyNarrower topic: Elivaldogene autotemcel is a specific gene therapy developed for selected cerebral cases.

• Canavan diseaseRelated: Experimental approaches aim to restore ASPA function or reduce harmful metabolite accumulation.

• Epidermolysis bullosaRelated: Clinical approaches aim to restore functional skin proteins in selected EB forms.

• Usher syndromeRelated: Researchers are investigating gene-based treatments for specific Usher syndrome subtypes.