ArticleHuman gene therapy. Clinical development2016
Investor Outlook: Rising from the Ashes; GSK's European Approval of Strimvelis for ADA-SCID.
Article in Human gene therapy. Clinical development, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
15 citing papers in PubMed, 44 citations in OpenAlex.
- Clinical hematopoietic stem cell-based gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Development of Lentiviral Packaging Cells and Scale Up of Production to Meet the Growing Demand in Cell and Gene Therapy.Current gene therapy · 2025Review
- Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications.Advanced drug delivery reviews · 2024Review
- Advancements in Hematopoietic Stem Cell Gene Therapy: A Journey of Progress for Viral Transduction.Cells · 2024Review
- Recent developments in gene therapy research in India.Journal of biosciences · 2024Review
- Reviving a Classic Antigen with a Cutting-Edge Approach: Nanobodies for HER2+ Breast Cancer.Pharmaceuticals (Basel, Switzerland) · 2023Review
- The Protective A673T Mutation of Amyloid Precursor Protein (APP) in Alzheimer's Disease.Molecular neurobiology · 2021Review
- Engineering precision therapies: lessons and motivations from the clinic.Synthetic biology (Oxford, England) · 2021Review
- Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB.Pharmaceutics · 2020Review
- Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality.Molecular therapy. Methods & clinical development · 2019Article
- Stimulus-responsive viral vectors for controlled delivery of therapeutics.Journal of controlled release : official journal of the Controlled Release Society · 2017Review
- Clinical development of CAR T cells-challenges and opportunities in translating innovative treatment concepts.EMBO molecular medicine · 2017Review
- Gene therapy research in Asia.Gene therapy · 2017Review
- Patient-driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis.EMBO molecular medicine · 2017Article
- Toll-like receptor 2 promiscuity is responsible for the immunostimulatory activity of nucleic acid nanocarriers.Journal of controlled release : official journal of the Controlled Release Society · 2017Article
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
GlaxoSmithKline's (GSK) and partner San Raffaele Telethon Institute for Gene Therapy's recent positive European approval for Strimvelis for treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) represents the second EU-approved gene therapy and the first γ-retrovirus and first ex vivo gene therapy. In this article we discuss the significance and implications of this historic approval for the broader gene therapy field.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.