gene-therapy

Documents about gene-therapy

• Raising Gene Therapy for Unmet Medical Needs in Japan#70404J-STAGE (Japan)
• Gene Therapy for Haemophilia A.#846589ClinicalTrials.gov
• Gene Therapy for Achromatopsia (CNGA3)#849504ClinicalTrials.gov
• AAVrh10-PCCA Gene Therapy for Propionic Acidemia#914594ClinicalTrials.gov
• Canavan-Single Patient IND#966495ClinicalTrials.gov
• Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy#308281HAL (France)
• Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C#910356ClinicalTrials.gov
• Pathing the way from regulatory approval to market access for gene therapy products (GTPs): An integrative review in the US, EU5, Japan and China#5235Europe PMC
• From gene-discovery to gene-tailored clinical management: 25 years of research in channelopathies and cardiomyopathies#294755HAL (France)
• Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy#966498ClinicalTrials.gov
• Appearance-related stigma and its implications for genetic counselling practice#256194Springer Nature OA
• A Study of EH002 Gene Therapy for Otoferlin Gene Mutation-mediated Hearing Loss#805659ClinicalTrials.gov
• Association Between the Effect of Glucocorticoid Pulse Therapy on Neuromyelitis Optica (NMO) and Gene Polymorphism#888752ClinicalTrials.gov
• Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A#979992ClinicalTrials.gov
• New Gene Therapy Strategy for β-Thalassemia.#266972NCBI PubMed Central
• Gene therapy for dystrophic epidermolysis bullosa#619767Deutsche Nationalbibliothek
• ASC618 Gene Therapy in Hemophilia A Patients#883214ClinicalTrials.gov
• Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease#841368ClinicalTrials.gov
• EndocardialVascularEndothelialGrowth Factor D(VEGF-D)Gene Therapy for the Treatment of Severe Coronary Heart Disease#835132ClinicalTrials.gov
• Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy#866774ClinicalTrials.gov
• Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector#827999ClinicalTrials.gov
• Host determinants of exo-rAAV production revealed by extended gene expression analysis in HEK293 cells.#928216Repisalud
• ZR001 Chemogenetics Gene Therapy Study in Patients With Parkinson's Disease#874868ClinicalTrials.gov
• Phase 1/2 Clinical Trial of LY3884963 in Patients With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)#991708ClinicalTrials.gov
• Innovative Contracting for Gene Therapies: Current Landscape and Perspectives on the Future of Gene Therapy Financing in the USA.#138615NCBI PubMed Central
• Gene Therapy for B-Cell Non-Hodgkin Lymphoma Using CD19 CAR Gene Transduced T Lymphocytes#429754ClinicalTrials.gov
• A Study of Talimogene Laherparepvec in Stage IIIc and Stage IV Malignant Melanoma#878547ClinicalTrials.gov
• THERAPEUTIC GENOME EDITING FOR HEREDITARY CONNECTIVE TISSUE DISORDERS#929422ScholarlyCommons
• ADVM-022 Intravitreal Gene Therapy for DME#217173ClinicalTrials.gov
• Activity-dependent antioxidant gene therapy protects against epileptogenesis#311544Europe PMC
• Data Management and Sharing Plan for: Lentiviral Gene Therapy of Hemophilia B#431904UNC Dataverse Dataverse OAI Archive
• Ecological Models for Gene Therapy. I. Models for Intraorganismal Ecology#761143PhilArchive
• Axonopathy in Duchenne Muscular Dystrophy limits microdystrophin gene therapy efficacy#779347bioRxiv / medRxiv
• Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity#176852HAL (France)
• Size specific transfection to mammalian cells by micropillar array electroporation#957651Louisiana Tech Digital Commons
• Study of an AAV Mediated Dual-Payload Gene Therapy in Patients With High Grade Glioma#801828ClinicalTrials.gov
• Fatal thrombotic microangiopathy case following adeno-associated viral SMN gene therapy#254049HAL (France)
• Gene-guided Warfarin for Anticoagulation Therapy#897908ClinicalTrials.gov
• A Gaucher Disease Gene Therapy Trial With FLT201#916919ClinicalTrials.gov
• Enhancing regulatory T cell therapy with gene editing / Viktor Glaser#1038086Deutsche Nationalbibliothek
• Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations#804093ClinicalTrials.gov
• Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD)#870571ClinicalTrials.gov
• An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)#916692ClinicalTrials.gov
• Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III#994354ClinicalTrials.gov
• Recombinant Adenoviral Human p53 Gene in Treatment of Malignant Pleural Effusion#804723ClinicalTrials.gov
• Follow-up of Breast Cancer and Multiple Myeloma Patients Previously Enrolled in NIH Gene Therapy Studies#811289ClinicalTrials.gov
• Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO)#842415ClinicalTrials.gov
• Effect of deoxyribonuclease I therapy on free circulating DNA level and gene methylation profile changes in patients with endometriosis: Proof of concept and preliminary data#460866HAL (France)
• Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)#995492ClinicalTrials.gov
• Long-Term Follow up of Patients Undergoing Hematopoietic Stem Cell Transplantation, Cellular Therapy, or Gene Therapy#448261ClinicalTrials.gov