Gene Therapies and Enhancement
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Gene Therapies and Enhancement
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Gene Therapies and Enhancement
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5.5SyntheticBiology5.4Science ofthe Originsof Life5.3FutureEconomics5.2Future ofEducation5.1ComplexSystemsScience4.4Democracy-affirming Technologies4.1Science-basedDiplomacy4.2Advances inScience Diplomacy4.3Digital Technologiesand Conflict3.7InfectiousDiseases3.6Solar RadiationModification3.5OceanStewardship3.4SpaceResources3.3Future FoodSystems3.2WorldSimulation3.1Decarbonisation2.6FutureTherapeutics2.5Organoids2.4ConsciousnessAugmentation2.3RadicalHealthExtension2.2HumanApplicationsof GeneticEngineering2.1CognitiveEnhancement1.6CollectiveIntelligence1.5AugmentedReality1.4BiologicalComputing1.3Brain-inspiredComputing1.2QuantumTechnologies1.1AdvancedAIHIGHEST ANTICIPATIONPOTENTIAL
5.5SyntheticBiology5.4Science ofthe Originsof Life5.3FutureEconomics5.2Future ofEducation5.1ComplexSystemsScience4.4Democracy-affirming Technologies4.1Science-basedDiplomacy4.2Advances inScience Diplomacy4.3Digital Technologiesand Conflict3.7InfectiousDiseases3.6Solar RadiationModification3.5OceanStewardship3.4SpaceResources3.3Future FoodSystems3.2WorldSimulation3.1Decarbonisation2.6FutureTherapeutics2.5Organoids2.4ConsciousnessAugmentation2.3RadicalHealthExtension2.2HumanApplicationsof GeneticEngineering2.1CognitiveEnhancement1.6CollectiveIntelligence1.5AugmentedReality1.4BiologicalComputing1.3Brain-inspiredComputing1.2QuantumTechnologies1.1AdvancedAIHIGHEST ANTICIPATIONPOTENTIAL

Sub-Field:

2.2.2Gene Therapies and Enhancement

    CRISPR-Cas9, though not even a decade old, is now the most widely used gene editing technology in the world. It is not only powerful, allowing multiple edits with a single manipulation, it is also widely accessible.8 What’s more, the technology is constantly advancing. In 2020, scientists reported on a successful clinical trial of an ex vivo CRISPR-based cancer immunotherapy on four patients with advanced melanoma and metastatic sarcoma, for example.9 In 2022, a trial of CRISPR-based treatment for acute myeloid leukaemia began. CRISPR has also shown promise in treating sickle cell anaemia and beta-thalassemia, and retinitis pigmentosa.

    Nevertheless, CRISPR-Cas9 has some important limitations as a human genome editor that may make other alternatives more attractive in the long term. The compound’s large size makes it more difficult to deliver into the cell than alternatives like zinc finger nucleases (ZNFs) and TALENs. CRISPR-Cas9 also seems to be more likely to trigger immune reactions.10

    Gene-based therapy and enhancement is therefore awaiting technological developments. While TALENS has been used to treat otherwise incurable childhood leukemia and ZNF has been used to treat Hunter’s syndrome in vivo, for the moment, these alternatives to CRISPR-Cas9 are less popular, since they are harder to use. Next-generation genome editors, such as mobile genetic elements, are already being used in the lab, and the future will probably see a combination of these technologies used, depending on the application. The boundary between what is considered therapy and enhancement is already somewhat blurred. Once in vivo genome edits become easy, cheap and mainstream, the line will become even more blurred, as will lines that demarcate prevention from treatment.

    Future Horizons:

    ×××

    5-yearhorizon

    Ex vivo and in vivo therapies advance

    Several large-scale stage III clinical trials for ex vivo therapies take place. Some ex vivo therapies are commercially available for some cancers and blood diseases. Early-stage clinical trials use in vivo editing techniques, targeting easily accessible tissues such as the cervix, the eye, or the liver. CRISPR corrects for mitochondrial genetic disease with in vitro fertilisation. Next-generation, novel genome editors appear on the stage.

    10-yearhorizon

    Safer genetic editing further blurs boundaries between therapy and prevention

    Somatic cell engineering (i.e. no germline) allows in-human treatment of conditions caused by the malfunction of several genes, such as cancer, diabetes, and cardiovascular diseases, and other conditions related to ageing. Human germline editing is used to prevent monogenic diseases, i.e. genetic diseases caused by a mutation on a single gene, such as cystic fibrosis, Duchenne type muscular dystrophy, and Huntington’s disease. We begin to see scattered preventative applications for preventive purposes.

    25-yearhorizon

    The boundaries between therapy and enhancement are eroded

    Human germline editing is mainstream, and we learn to engineer new sensory capacities for humans. Genome-editing conveys a higher resistance to radiation and becomes key for space travel. We use gene technologies to correct, slow down or even reverse processes linked to ageing.

    Gene Therapies and Enhancement - Anticipation Scores

    How the experts see this field in terms of the expected time to maturity, transformational effect across science and industries, current state of awareness among stakeholders and its possible impact on people, society and the planet. See methodology for more information.

    GESDA Best Reads and Key Resources

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    What’s new in clinical CRISPR? - Nature Medicine

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    28th Jan 2021
    In the past year, there has been more CRISPR news than the awarding of the Nobel Prize in Chemistry to Jennifer Doudna and Emmanuelle Charpentier. Gene editing has continued its forward momentum in major ways, and scientists are beginning to see results.