Works matching DE "GENE therapy"
Results: 5000
Advances in cryo-electron microscopy (cryoEM) for structure-based drug discovery.
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- Expert Opinion on Drug Discovery, 2025, v. 20, n. 2, p. 163, doi. 10.1080/17460441.2025.2450636
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Late effects following hematopoietic cell transplantation for severe combined immunodeficiency: critical factors and therapeutic options.
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- Expert Review of Clinical Immunology, 2025, v. 21, n. 1, p. 73, doi. 10.1080/1744666X.2024.2402948
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Invited Speakers.
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- Clinical & Experimental Ophthalmology, 2025, v. 53, n. 1, p. 13, doi. 10.1111/ceo.14481
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ASCPT News.
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- Clinical Pharmacology & Therapeutics, 2025, v. 117, n. 4, p. 874, doi. 10.1002/cpt.3594
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ASCPT News.
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- Clinical Pharmacology & Therapeutics, 2025, v. 117, n. 3, p. 614, doi. 10.1002/cpt.3551
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- Article
Highlighted Articles.
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- Clinical Pharmacology & Therapeutics, 2025, v. 117, n. 3, p. 612, doi. 10.1002/cpt.3548
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- Article
ASCPT News.
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- Clinical Pharmacology & Therapeutics, 2025, v. 117, n. 2, p. 326, doi. 10.1002/cpt.3522
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- Article
Loss‐of‐Function of CLMP Is Associated With Congenital Short Bowel Syndrome and Impaired Intestinal Development.
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- Clinical Genetics, 2025, v. 107, n. 4, p. 413, doi. 10.1111/cge.14678
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Is It Possible to Regenerate the Underactive Detrusor? Part 1. Molecular and Stem Cell Therapies Targeting the Urinary Bladder and Neural Axis ‐ ICI‐RS 2024.
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- Neurourology & Urodynamics, 2025, v. 44, n. 3, p. 577, doi. 10.1002/nau.25597
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Diagnosis and Treatment of Alpha Thalassemia Major.
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- Hemoglobin, 2025, v. 49, n. 1, p. 3, doi. 10.1080/03630269.2024.2432899
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Sickle cell anaemia therapy in 2025.
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- British Journal of Haematology, 2025, v. 206, n. 3, p. 842, doi. 10.1111/bjh.19933
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Development of a macrophage polarization-modulating therapeutic agent for osteoarthritis treatment.
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- Journal of Orthopaedic Surgery & Research, 2025, v. 20, n. 1, p. 1, doi. 10.1186/s13018-025-05679-2
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Self‐Adaptive Activation of DNAzyme Nanoassembly for Synergistically Combined Gene Therapy.
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- Angewandte Chemie, 2025, v. 137, n. 5, p. 1, doi. 10.1002/ange.202417363
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The First Fifty Years of Stem Cell Transplantation in Severe Combined Immunodeficiency (SCID).
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- Acta Medica Iranica, 2024, v. 62, n. 6, p. 297
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Impact of liver fibrosis on AAV-mediated gene transfer to mouse hepatocytes.
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- Nature Communications, 2025, v. 16, n. 1, p. 1, doi. 10.1038/s41467-025-57382-9
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Approach to the Child and Adolescent With Adrenal Insufficiency.
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- Journal of Clinical Endocrinology & Metabolism, 2025, v. 110, n. 3, p. 863, doi. 10.1210/clinem/dgae564
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Prediction of genome base-editing efficiency and outcomes based on machine learning: A deep review.
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- Journal of Biotech Research, 2024, v. 18, p. 183
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THE ROLE OF GENE THERAPY IN ORTHODONTICS.
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- Guident, 2025, v. 18, n. 3, p. 26
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Global research dynamics in urea cycle disorders: a bibliometric study highlighting key players and future directions.
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- Orphanet Journal of Rare Diseases, 2025, v. 20, n. 1, p. 1, doi. 10.1186/s13023-025-03625-3
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Immunotherapy of Pancreatic Cancer Mediated by KRAS Mutant Peptides.
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- Journal of East China University of Science & Technology, 2025, v. 51, n. 1, p. 60, doi. 10.14135/j.cnki.1006-3080.20240319001
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The Benefits of Sustaining High Factor VIII Levels in People With Hemophilia A.
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- American Journal of Managed Care, 2025, p. S15, doi. 10.37765/ajmc.2025.89703
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Development of CAR-T cell therapy for NF1/SWN-related nerve sheath tumor treatment.
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- Acta Neuropathologica Communications, 2025, v. 13, n. 1, p. 1, doi. 10.1186/s40478-025-01965-6
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The role of telomere and telomerase in cancer and novel therapeutic target: narrative review.
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- Frontiers in Oncology, 2025, p. 1, doi. 10.3389/fonc.2025.1542930
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Membrane Filtration of Nanoscale Biomaterials: Model System and Membrane Performance Evaluation for AAV2 Viral Vector Clarification and Recovery.
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- Nanomaterials (2079-4991), 2025, v. 15, n. 4, p. 310, doi. 10.3390/nano15040310
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Genetic Insights and Clinical Implications of NEU1 Mutations in Sialidosis.
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- Genes, 2025, v. 16, n. 2, p. 151, doi. 10.3390/genes16020151
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Therapeutic Advances in Bladder Preservation for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer.
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- Cancers, 2025, v. 17, n. 4, p. 636, doi. 10.3390/cancers17040636
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Molecular and Digital Innovation in Pediatric Surgery.
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- Molecular Frontiers Journal, 2024, v. 8, n. 1/2, p. 37, doi. 10.1142/S2529732524500032
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- Article
Regional Gene Therapy for Bone Tissue Engineering: A Current Concepts Review.
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- Bioengineering (Basel), 2025, v. 12, n. 2, p. 120, doi. 10.3390/bioengineering12020120
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Toxicity Evaluation of Sulfobetainized Branched Polyethyleneimine via Antibacterial and Biocompatibility Assays.
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- Toxics, 2025, v. 13, n. 2, p. 136, doi. 10.3390/toxics13020136
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Role of the AIM2 Inflammasome in Cancer: Potential Therapeutic Strategies.
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- Biomedicines, 2025, v. 13, n. 2, p. 395, doi. 10.3390/biomedicines13020395
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Looking to the Future of Viral Vectors in Ocular Gene Therapy: Clinical Review.
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- Biomedicines, 2025, v. 13, n. 2, p. 365, doi. 10.3390/biomedicines13020365
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Consecutive Affinity and Ion-Exchange Chromatography for AAV9 Vectors Purification.
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- Biomedicines, 2025, v. 13, n. 2, p. 361, doi. 10.3390/biomedicines13020361
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Comparative analysis of AAV serotypes for transduction of olfactory sensory neurons.
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- Frontiers in Neuroscience, 2025, p. 1, doi. 10.3389/fnins.2025.1531122
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Frontier applications of retinal nanomedicine: progress, challenges and perspectives.
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- Journal of Nanobiotechnology, 2025, v. 23, n. 1, p. 1, doi. 10.1186/s12951-025-03095-6
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Emerging Gene Therapy Based on Nanocarriers: A Promising Therapeutic Alternative for Cardiovascular Diseases and a Novel Strategy in Valvular Heart Disease.
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- International Journal of Molecular Sciences, 2025, v. 26, n. 4, p. 1743, doi. 10.3390/ijms26041743
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Tropism of the AAV6.2 Vector in the Murine Retina.
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- International Journal of Molecular Sciences, 2025, v. 26, n. 4, p. 1580, doi. 10.3390/ijms26041580
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Gene Therapy: An Historical Overview for Familial Hearing Loss.
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- International Journal of Molecular Sciences, 2025, v. 26, n. 4, p. 1469, doi. 10.3390/ijms26041469
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From Origin to the Present: Establishment, Mechanism, Evolutions and Biomedical Applications of the CRISPR/Cas-Based Macromolecular System in Brief.
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- Molecules, 2025, v. 30, n. 4, p. 947, doi. 10.3390/molecules30040947
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BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease.
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- Science Translational Medicine, 2025, v. 17, n. 787, p. 1, doi. 10.1126/scitranslmed.ads0539
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Diagnosis and Treatment of Neonatal Diabetes Caused by ATP-Channel Mutations: Genetic Insights, Sulfonylurea Therapy, and Future Directions.
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- Children, 2025, v. 12, n. 2, p. 219, doi. 10.3390/children12020219
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Analysis of Hepatic Lentiviral Vector Transduction: Implications for Preclinical Studies and Clinical Gene Therapy Protocols.
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- Viruses (1999-4915), 2025, v. 17, n. 2, p. 276, doi. 10.3390/v17020276
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Adeno-Associated Virus Vectors: Principles, Practices, and Prospects in Gene Therapy.
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- Viruses (1999-4915), 2025, v. 17, n. 2, p. 239, doi. 10.3390/v17020239
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MANAGING THE EVOLUTION OF HEALTHCARE: INTEGRATING GENETIC ENGINEERING INTO MODERN MEDICINE.
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- German International Journal of Modern Science / Deutsche Internationale Zeitschrift für Zeitgenössische Wissenschaft, 2025, n. 98, p. 5, doi. 10.5281/zenodo.14910698
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Correction to: Nadofaragene firadenovec in high-risk Bacillus Calmette Guérin unresponsive non-muscle invasive bladder cancer: a profile of its use.
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- 2024
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- Correction Notice
Nadofaragene firadenovec in high-risk Bacillus Calmette Guérin unresponsive non-muscle invasive bladder cancer: a profile of its use.
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- Drugs & Therapy Perspectives, 2024, v. 40, n. 1, p. 1, doi. 10.1007/s40267-024-01045-0
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An overview of recent US-approved gene therapies for Duchenne muscular dystrophy and their respective clinical development programs.
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- Drugs & Therapy Perspectives, 2023, v. 39, n. 4, p. 156, doi. 10.1007/s40267-023-00986-2
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It's Just Different in the Country: Postnatal Depression and Group Therapy in a Rural Setting.
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- Social Work in Health Care, 2002, v. 34, n. 3/4, p. 333, doi. 10.1300/J010v34n03_06
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When size matters: A novel compact Cas12a variant for in vivo genome editing.
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- PLoS Biology, 2024, v. 22, n. 7, p. 1, doi. 10.1371/journal.pbio.3002637
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Engineering of a compact, high-fidelity EbCas12a variant that can be packaged with its crRNA into an all-in-one AAV vector delivery system.
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- PLoS Biology, 2024, v. 22, n. 5, p. 1, doi. 10.1371/journal.pbio.3002619
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Inhibited Apoptosis of C<sub>2</sub>C<sub>12</sub> Myoblasts by a Eupatorium chineuse var. simplicifolium Root Extract.
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- Bioscience, Biotechnology & Biochemistry, 2013, v. 77, n. 10, p. 2134, doi. 10.1271/bbb.130333
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