December 11, 2019 article Open access Gene therapy for severe combined immunodeficiencies and beyond The Journal of Experimental Medicine DOI: 10.1084/jem.20190607 Full text (OA) OpenAlex Authors 0 Author list not loaded yet. Cited by 5 stored of 104 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 Paying for precision: funding approaches for N-of-1 trials of individualized gene targeted therapies 2026 Orphanet Journal of Rare Diseases article Economics, Econometrics and Finance Pharmaceutical Economics and Policy Open access 3 citations Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells 2024 Biomedicines article Biochemistry, Genetics and Molecular Biology Virus-based gene therapy research Open access 5 citations Advanced Therapy Medicinal Products: Clinical, Non-clinical, and Quality Considerations 2024 Springer eBooks book-chapter Medicine Biomedical Ethics and Regulation 0 citations Precise homology-directed installation of large genomic edits in human cells with cleaving and nicking high-specificity Cas9 variants 2023 Nucleic Acids Research article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access 19 citations Modulating mutational outcomes and improving precise gene editing at CRISPR-Cas9-induced breaks by chemical inhibition of end-joining pathways 2023 Cell Reports article Biochemistry, Genetics and Molecular Biology CRISPR and Genetic Engineering Open access cited by 5 patents 75 citations 5 results References 0