August 15, 2022 article Ensuring a future for gene therapy for rare diseases Nature Medicine DOI: 10.1038/s41591-022-01934-9 OpenAlex Authors 0 Author list not loaded yet. Cited by 6 stored of 57 Search Sort Most cited Newest Oldest Patent citations Title Any typearticle review book-chapter conference-paper preprint dissertation book dataset other Any fieldAgricultural and Biological Sciences Arts and Humanities Biochemistry, Genetics and Molecular Biology Business, Management and Accounting Chemical Engineering Chemistry Computer Science Decision Sciences Dentistry Earth and Planetary Sciences Economics, Econometrics and Finance Energy Engineering Environmental Science Health Professions Immunology and Microbiology Materials Science Mathematics Medicine Neuroscience Nursing Pharmacology, Toxicology and Pharmaceutics Physics and Astronomy Psychology Social Sciences Veterinary Open access Advancing the Integration of ‘Basic/Fundamental’ and Translational Cellular and Gene Therapy Science within the EBMT: Accelerating the Pathway to Progress 2025 Bone Marrow Transplantation article Medicine Hematopoietic Stem Cell Transplantation Open access 1 citations Navigating disruption in the PID landscape: embracing opportunities and anticipating threats in the next ten years 2025 Frontiers in Immunology article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders Open access 2 citations Advances in gene therapy for inborn errors of immunity 2023 Current Opinion in Allergy and Clinical Immunology article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access 23 citations The Relevance of Advanced Therapy Medicinal Products in the Field of Transplantation and the Need for Academic Research Access: Overcoming Bottlenecks and Claiming a New Time 2023 Transplant International article Medicine Biomedical Ethics and Regulation Open access 16 citations Prospective Newborn Screening for SCID in Germany: A First Analysis by the Pediatric Immunology Working Group (API) 2023 Journal of Clinical Immunology article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders Open access 54 citations Access to gene therapy for rare diseases when commercialization is not fit for purpose 2023 Nature Medicine article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access 50 citations 6 results References 2 Search Sort Most cited Newest Oldest Patent citations Title Any typearticle review book-chapter conference-paper preprint dissertation book dataset other Any fieldAgricultural and Biological Sciences Arts and Humanities Biochemistry, Genetics and Molecular Biology Business, Management and Accounting Chemical Engineering Chemistry Computer Science Decision Sciences Dentistry Earth and Planetary Sciences Economics, Econometrics and Finance Energy Engineering Environmental Science Health Professions Immunology and Microbiology Materials Science Mathematics Medicine Neuroscience Nursing Pharmacology, Toxicology and Pharmaceutics Physics and Astronomy Psychology Social Sciences Veterinary Open access Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort 2021 Journal of Allergy and Clinical Immunology article Immunology and Microbiology Immunodeficiency and Autoimmune Disorders Open access 106 citations Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook 2020 Nature Reviews Drug Discovery article Mathematics Statistical Methods in Clinical Trials 30 citations 2 results